USA > History > 2010 > Health (III)

Judge Rejects City Law

on Antismoking Posters

December 29, 2010
The New York Times
By ANAHAD O’CONNOR

A federal judge on Wednesday struck down a New York City law that would have forced all bodegas and convenience stores to post gruesome images of diseased lungs, brains and teeth in the shops to discourage people from buying cigarettes.

In a 13-page ruling, Judge Jed S. Rakoff of United States District Court in Manhattan wrote that while the law was well intentioned, it violated federal law since only the federal government had the authority to regulate cigarette warnings and advertisements.

“Even merchants of morbidity are entitled to the full protection of the law,” Judge Rakoff wrote, “for our sake as well as theirs.”

The decision puts an end — at least for now— to the city’s plan to have the placards displayed beside cash registers in more than 11,000 establishments across the city. While awaiting Judge Rakoff’s ruling, the city had agreed that it would postpone enforcement of its rule until this weekend.

Lawyers with the city’s Law Department said they planned to appeal the decision.

The city’s health department created the rule in late 2009. In June, the nation’s three biggest tobacco companies — Philip Morris, Lorillard and R. J. Reynolds — joined forces with the New York State Association of Convenience Stores in filing a lawsuit challenging the rule. In their suit, the tobacco companies and convenience stores said the rule violated the First Amendment rights of retailers who disagreed with the message, and breached a law stating that only the federal government can regulate cigarette warnings and advertising.

In his ruling, Judge Rakoff said that health officials had good reason to view smoking as a “public health threat,” citing smoking as the leading cause of preventable death in New York City and the rest of the country. “Within New York City, roughly 7,500 people die from smoking annually — more than from AIDS, homicide and suicide combined,” he wrote.

But Judge Rakoff also cited a federal law enacted in 1965, the Labeling Act, which gave the federal government exclusive authority over cigarette warnings. That law, he wrote, seeks to balance public and commercial interests: the federal government protects the public, but also sets clear and uniform cigarette regulations that protect “commerce and the national economy.”

In his ruling, Judge Rakoff pointed out that the Labeling Act also contained a provision forbidding any state laws from conflicting with the federal government’s policies on cigarette warnings and advertisements. That, he concluded, makes the city’s placard policy illegal.

Floyd Abrams, a lawyer who represented the convenience store association, said that even though the city had agreed not to enforce the rule until this weekend, many retail shops had put up the graphic placards anyway. He said he was “very pleased” with Judge Rakoff’s decision.

“It will allow the retail stores in New York to be freed of the obligation to put signs up urging customers not to buy their lawful products,” he said.

In a statement, the health department said that the city “strongly disagrees” with the ruling and that tobacco companies “trying to prevent these messages from being seen should be ashamed of themselves.”

“The city’s warning signs portray completely factual messages about the dangers of smoking,” the statement added. “They do so at the exact moment when smokers are making decisions about purchasing tobacco. We believe it is the city’s responsibility to help smokers quit and to protect children from the harmful effects of tobacco smoke.”

    Judge Rejects City Law on Antismoking Posters, NYT, 29.12.2010, http://www.nytimes.com/2010/12/30/nyregion/30smoking.html

Enlisting the Dying for Clues to Save Others

December 26, 2010
The New York Times
By AMY HARMON

LOS ANGELES — They had told him on his last visit: the experimental drug that had so miraculously melted his tumors was no longer working. His legs were swollen, the melanoma erupting in angry black lumps. The patient, a computer consultant in his 40s, had little time left.

And now the man’s doctor, Roger Lo of the cancer center at the University of California, Los Angeles, was calling to ask whether they could harvest a slice of one of his resurgent tumors for research he would almost certainly not be alive to benefit from. He would need to fly to Los Angeles from Northern California at his own expense, subject himself to an injection of anesthetic and the slight risk of infection, and spend yet another afternoon in the hospital.

“I was hoping,” Dr. Lo said that day last spring, “you would come in for a biopsy.”

The hope lies in a new breed of cancer drugs that work by blocking the particular genetic defect driving an individual tumor’s out-of-control growth — in the case of Dr. Lo’s patient, a single overactive protein. If researchers can pinpoint which new genetic alteration is driving the cancer when it evades the blockade — as it nearly always does — similarly tailored drugs may be able to hold it off for longer. The crucial evidence resides in the tumor cells of patients who, like Dr. Lo’s, have relapsed.

But the need to ask those who know their time is short to undergo another invasive procedure in the name of science is just one obstacle to what many oncologists see as the best chance to give future cancer patients a more permanent reprieve.

A regulatory process that can take years to approve a drug for sale means that instead of thousands of patients to draw on, only the few hundred who receive the drug through clinical trials are available for such research. Ethical review boards frown on any procedure that exposes patients to unnecessary risk, like the rupturing of a blood vessel or puncturing of a lung. Some hard-won tumor samples prove unsuitable for research.

Then there is the question of who will pay for the biopsies, which cost as much as $5,000 and typically cannot be billed to insurance. Dr. Lo, for one, covered the costs when there was no other means to pay.

As drugs tailored to the genetics of particular tumors make their way through early clinical trials, similar quests to improve on them are being undertaken by researchers in the various forms of a disease that kills more than a half-million Americans and millions more people worldwide every year.

Dr. Lo’s quest to understand how melanoma forges its resistance to the drug PLX4032, made by Roche, illustrates the Herculean effort required to take even a baby step toward a cure for cancer. Finding a single clue that could lead to the testing of one new drug that might help a small fraction of patients took two long years.

But it also shows how such progress emerges, from a complex mix of academic ambition, collaboration and competition among scientists, and, especially, the willing participation of dying patients.

When the man Dr. Lo had called arrived in his office a week later, tumors covered his legs from the bottom of his feet to his groin. Some of them were infected, their odor so overwhelming that the doctor put on a mask before administering an anesthetic and cutting into his left thigh.

A Researcher’s Trials

Dr. Roger Lo, 38, was an unlikely player in the scramble to improve on the Roche drug, which had been given to only a handful of patients when its successes began to grab the field’s attention in late 2008.

An assistant professor in dermatology, he had started his own laboratory only a few months earlier and had been advised by senior colleagues to avoid high-risk projects until he secured a steady source of financial support.

But like others in the field, he was galvanized by watching melanoma patients respond to the Roche drug, the first to reliably slow a disease that typically kills within a year of diagnosis, and rarely responds to chemotherapy.

Studying how to prevent a relapse, he argued in a grant proposal in early 2009, was “of paramount importance.”

Impressed by his drive, and equally eager to realize the full promise of the drug’s approach, Dr. Antoni Ribas, the melanoma oncologist running U.C.L.A.’s arm of the drug’s clinical trial, agreed to collaborate with Dr. Lo. Bigger, better-financed laboratories pursuing the same question might reach an answer first, Dr. Ribas warned.

But Dr. Lo had reason to hope the results would be published in a leading journal if he was the first to find the culprit responsible for reigniting the cancer, among the many possibilities. That could prompt drug companies to speed the development of a new therapy.

Lacking tumor samples from patients in the Roche trial, Dr. Lo instead sought to replicate the cancer’s resistance to the drug by feeding a steady diet of the drug to melanoma cells taken from three previous patients who had never received it. When the few cancer cells that survived the onslaught began to grow in their petri dishes, he used those, now resistant to the drug, to begin his search.

It could have been straightforward. Many researchers believed the answer would be that the gene whose mutation initially made the protein that drove the cancer’s uncontrolled growth had mutated again, as had happened in other cancers. In a few cases, a new drug tailored to the new mutations had lengthened remissions.

But Dr. Lo found no evidence of this. Nor did he find the smoking gun in several other genes linked to the growth of other cancers.

Instead, he began the painstaking process of measuring the activity of hundreds of proteins that might have driven the cancer’s uncontrolled growth. The experiments required modifying the levels of each protein in the drug-resistant cells, dosing them with the drug and checking every few hours to see how fast they were growing. With only two junior scientists and a technician in his laboratory, Dr. Lo performed much of the work himself.

Even so, he knew, nothing he found in the cells whose resistance he had artificially bred in the lab would matter unless he also found it in the patients who had relapsed.

One Patient’s Contribution

Those who wonder whether a single patient can help cancer research should know the case of Lee Reyes.

Thirty years old when his advanced melanoma was diagnosed in early 2008, Mr. Reyes was distraught at how little he had accomplished. Introverted and a perfectionist, he had dropped out of college and lived with his parents in Fresno, Calif. He cycled through video game systems, favoring the Xbox. He loved flying and thought about getting a helicopter pilot’s license, but never pursued it.

“For the better part of about 10 years I did close to nothing,” he said two years ago. “I just always felt I had so much time.”

One of Dr. Ribas’s first patients in the trial of the Roche drug, Mr. Reyes was selected because he was among the half of melanoma patients whose tumors carried the overactive protein the drug blocked. As it would for nearly every patient in the trial, the drug held his cancer at bay for several months. But as would happen with the others, his response did not last.

With his life at immediate risk because of a melanoma tumor that had metastasized to his heart, Mr. Reyes traveled to U.C.L.A. for surgery in May 2009, agreeing to let his tumor be used for research.

On Dr. Ribas’s instructions, a technician stood in Mr. Reyes’s surgery room and, as soon as the surgeon extracted the tumor, ran with it to the nearby laboratory to reduce the chance of exposure to contamination. To coax the cancer cells to thrive so that Dr. Lo could run them through a battery of tests, it was sliced up with sterile knives and deposited, in a flask with sugar solution, in an incubator.

“Let’s hope it grows,” Dr. Ribas said to Dr. Lo.

On a visit to Mr. Reyes’s room after the surgery, Dr. Ribas did not discuss the future with his patient. They both knew the options were limited. Instead, they talked of animals: Mr. Reyes’s affinity for monkeys — he was clutching a stuffed one from a hospital gift shop — and Dr. Ribas’s for sea otters.

When Mr. Reyes died a few months later, Dr. Ribas called his mother to offer his condolences, as is his custom. And then he told her something else.

“He said Lee is helping them,” Ellen Reyes told her husband.

Mr. Reyes’s cells were growing.

A Breakthrough

It would take months for Mr. Reyes’s cells to multiply to the numbers Dr. Lo needed to perform his tests. And that summer, the foundation he had hoped would finance his research judged his grant proposal “too ambitious” for a junior investigator.

But by late September 2009, using the laboratory cells he had earlier bred to resist the Roche drug, he had narrowed his search. The cancer’s new driver, he believed, was one of 42 proteins on the surface of the cell. A few weeks later, he closed in. An experiment that could detect all 42 found a single culprit, appearing as twin dots of black on the translucent background of the film: the resistant cells contained 10 times more of the protein than those that were still responding to the Roche drug in Dr. Lo’s petri dishes.

And, he noted with excitement, a drug designed to block that protein was already being prescribed for other cancers. Perhaps a solution for patients was available already.

But as he prepared to see whether Mr. Reyes’s cells bore out the observation, Dr. Lo tried to restrain his hopes.

“We have a likely candidate,” he told Dr. Ribas carefully.

It was probable, he reminded himself, that this protein was not the source of the cancer’s resistance in all patients. In fact, the cancer could reroute itself differently in every patient. Even if the theory was right, Mr. Reyes’s tumor might not reflect it.

In October, as the precious cells grew close to the number required for the experiment, Dr. Ribas received a box in the mail. In it was a stuffed sea otter, and a note from Mr. Reyes’s mother.

They had visited the Monterey Aquarium over the summer, she wrote. Mr. Reyes had bought the otter to take to Dr. Ribas on his next trip.

On a Saturday night in mid-November, Dr. Lo called Dr. Ribas. He was looking at the film showing the results of the experiment on Mr. Reyes’s cells. On the translucent background, the same twin dots showed black.

The patient’s cells, still living, harbored levels of the protein far higher than even Dr. Lo’s laboratory models.

“Toni,” he said. “You have to see this for yourself.”

The Scramble for Donors

To be confident that his find was not a fluke, Dr. Lo needed more samples from other patients. Their goal would be 10, he and Dr. Ribas agreed in late-night e-mail exchanges. The best would be “before” and “after” snapshots from patients at the beginning of their treatment and after they had relapsed. But those were in short supply.

Only 48 patients had been treated in the drug’s first trial, eight at each of six leading cancer centers. Many had not been biopsied when their cancer returned. Some of the biopsies had been sent to Roche, which had not yet shared its own research with the academic researchers.

Briefly, researchers at the six sites contemplated pooling the few samples in their possession, and sharing authorship of their results. But the tissues could be subjected to only so many tests before being used up. And success in academia can hinge on being listed as a paper’s lead author. When several group discussions failed to yield even a complete accounting of who had how many samples, researchers agreed to stay in touch, but go it separately.

Still, in late 2009, Dr. Ribas approached two of the oncologists he knew to ask if they would share samples with Dr. Lo.

“I think he really has something,” he told the two, Dr. Jeffrey Sosman and Dr. Grant McArthur.

Four tumor samples, two from each doctor, arrived a few days later by FedEx. One, from a retired math teacher in Tennessee, tested positive for high levels of Dr. Lo’s protein.

The same protein was hyperactive in another sample, which came from a Croatian patient of Dr. Ribas’s who had been commuting to Los Angeles for treatment. The patient had surgery in Croatia to remove a tumor in his abdomen and requested that it be sent to Dr. Ribas.

Like everyone treating the patients in the Roche trial, Drs. Lo and Ribas were growing increasingly tormented by the knowledge that they could not promise more than a few months’ reprieve to the patients enrolled in the drug trial.

They also knew that other researchers were close to publishing their own findings. One had given a presentation at a conference, and there was a rumor that his paper had been tentatively accepted by Nature, a premier science journal.

Over the first six months of this year, the doctors renewed their efforts to perform biopsies. Dr. Lo sent gentle e-mail reminders to the other oncologists with patients on the Roche drug, who might forget to ask or sidestep the difficult conversation.

He juggled his schedule to be available whenever the opportunity arose, borrowing exam rooms to squeeze patients in. He scraped exposed tumor off the neck of a dance teacher in her 60s, and sliced into the lower back of a mortgage broker in her 50s.

Roche’s rules for how biopsy samples would be stored in the next, larger trial of its drug made it impossible to perform many of the tests Dr. Lo wanted to try. So the U.C.L.A. doctors asked patients to sign a separate consent form, authorized by the university’s ethical review board, for research that would be conducted independent of the drug company.

There were some biopsies they did not get. One patient’s family volunteered an autopsy if it would help, but the timing made it impossible: Dr. Lo needed living tissue for the studies he was conducting. The doctors decided against asking another patient, a young mother whose tumor was in her liver. The risk of complications did not justify the benefit.

A prominent immunologist from San Diego was willing to subject himself to a biopsy of a tumor near his knee, but the U.C.L.A. surgeon turned him away on the operating table, judging it too painful to remove. Still, when the immunologist, Dr. Norman Klinman, underwent surgery in San Diego after relapsing, his son raced to deliver his tumor sample, strapped into the front seat of his car in a container of dry ice, to Dr. Ribas’s laboratory.

By early May, they had nine samples. Dr. Lo’s three lab workers had worked nearly every weekend for a year, and he lacked the money to hire more. Another of his grant proposals had been turned down because a reviewer said the Roche drug was too early in its testing to warrant a search for the cause of resistance to it.

When the machine they used to measure cell growth broke down that month and dozens of experiments had to be redone, Dr. Lo’s postdoctoral trainee mentioned a long-postponed trip to see relatives on the East Coast.

“Put it on hold,” Dr. Lo urged. “We’re almost there.”

An Elusive Target

When Dr. Lo submitted his paper to Nature last July, he had collected tumor samples from 12 patients. Of the four that contained his suspect protein, three of the patients had died by the time he had identified it in their biopsy.

But the last one came from a 54-year-old Canadian named Wes Coyle, who was alive, but barely, when Dr. Lo confirmed the protein’s presence through a biopsy of a tumor in his pelvis. It was the first time that, using his and Dr. Lo’s research, Dr. Ribas could try to help a patient who had relapsed. He warned Mr. Coyle’s sister Peggy Coyle Seaver, who was caring for her brother, that it was a long shot. But he prescribed the drug, made by Pfizer, that was known to block the protein in some other cancers.

For two weeks, Ms. Seaver reported to the doctor, her brother was able to eat again. Yet he died soon after that.

Another opportunity arose with a fifth patient who contributed tumor samples to Dr. Lo’s collection, an avid gardener in Los Angeles. Her sample carried a gene mutation that suggested a second possible escape route for the cancer. But on a clinical trial for a drug specifically designed to block it, she quickly deteriorated.

Most likely, the two doctors knew, single drugs would not alone block the resurgent cancer, even if they were hitting the right targets.

“Damn it!” Dr. Ribas exploded, calling Dr. Lo when he saw the tumors growing on her scans. Of the 12 patient samples, Dr. Lo had identified the likely source of the cancer’s resurgence in five. In August, with a grant he received from the Melanoma Research Alliance, along with researchers at other institutions, Dr. Lo began to probe the rest.

And in late November, after requesting another round of experiments, Nature published his paper, along with one from a competing researcher.

Ms. Seaver scanned the copy Dr. Ribas sent her, her breath catching when she recognized her brother, listed in a table as the 54-year-old male who had a 78-day response. “It feels like Wesley is still alive,” she told her husband.

Brian Lewis, a paramedic whose wife Dr. Lo had decided against asking for a liver biopsy, read about his paper on the Internet, and contacted the doctor to ask whether providing one now would help him. His wife was doing poorly, but they had two young children, he wrote, “and I would hate to see them fight the same battle their mom is fighting.”

A researcher at Memorial Sloan-Kettering Cancer Center consulted his own batch of five tumor samples from patients who had relapsed. One of them, he told colleagues on a conference call, had the second mutation Dr. Lo had found.

In early December, Dr. Ribas visited Roche’s offices in San Francisco. The data in the paper, he argued, made a case for clinical trials of combinations of drugs the company was already developing. Researchers would need to take biopsies from patients in those trials, too, they all agreed. Because the cancer, even if blocked a second time, might find another way through.

“We have a lot more to do,” Dr. Lo tells his laboratory staff every day.

    Enlisting the Dying for Clues to Save Others, NYT, 26.12.2010, http://www.nytimes.com/2010/12/27/health/research/27trials.html

Obama Returns to End-of-Life Plan That Caused Stir

December 25, 2010
The New York Times
By ROBERT PEAR

WASHINGTON — When a proposal to encourage end-of-life planning touched off a political storm over “death panels,” Democrats dropped it from legislation to overhaul the health care system. But the Obama administration will achieve the same goal by regulation, starting Jan. 1.

Under the new policy, outlined in a Medicare regulation, the government will pay doctors who advise patients on options for end-of-life care, which may include advance directives to forgo aggressive life-sustaining treatment.

Congressional supporters of the new policy, though pleased, have kept quiet. They fear provoking another furor like the one in 2009 when Republicans seized on the idea of end-of-life counseling to argue that the Democrats’ bill would allow the government to cut off care for the critically ill.

The final version of the health care legislation, signed into law by President Obama in March, authorized Medicare coverage of yearly physical examinations, or wellness visits. The new rule says Medicare will cover “voluntary advance care planning,” to discuss end-of-life treatment, as part of the annual visit.

Under the rule, doctors can provide information to patients on how to prepare an “advance directive,” stating how aggressively they wish to be treated if they are so sick that they cannot make health care decisions for themselves.

While the new law does not mention advance care planning, the Obama administration has been able to achieve its policy goal through the regulation-writing process, a strategy that could become more prevalent in the next two years as the president deals with a strengthened Republican opposition in Congress.

In this case, the administration said research had shown the value of end-of-life planning.

“Advance care planning improves end-of-life care and patient and family satisfaction and reduces stress, anxiety and depression in surviving relatives,” the administration said in the preamble to the Medicare regulation, quoting research published this year in the British Medical Journal.

The administration also cited research by Dr. Stacy M. Fischer, an assistant professor at the University of Colorado School of Medicine, who found that “end-of-life discussions between doctor and patient help ensure that one gets the care one wants.” In this sense, Dr. Fischer said, such consultations “protect patient autonomy.”

Opponents said the Obama administration was bringing back a procedure that could be used to justify the premature withdrawal of life-sustaining treatment from people with severe illnesses and disabilities.

Section 1233 of the bill passed by the House in November 2009 — but not included in the final legislation — allowed Medicare to pay for consultations about advance care planning every five years. In contrast, the new rule allows annual discussions as part of the wellness visit.

Elizabeth D. Wickham, executive director of LifeTree, which describes itself as “a pro-life Christian educational ministry,” said she was concerned that end-of-life counseling would encourage patients to forgo or curtail care, thus hastening death.

“The infamous Section 1233 is still alive and kicking,” Ms. Wickham said. “Patients will lose the ability to control treatments at the end of life.”

Several Democratic members of Congress, led by Representative Earl Blumenauer of Oregon and Senator John D. Rockefeller IV of West Virginia, had urged the administration to cover end-of-life planning as a service offered under the Medicare wellness benefit. A national organization of hospice care providers made the same recommendation.

Mr. Blumenauer, the author of the original end-of-life proposal, praised the rule as “a step in the right direction.”

“It will give people more control over the care they receive,” Mr. Blumenauer said in an interview. “It means that doctors and patients can have these conversations in the normal course of business, as part of our health care routine, not as something put off until we are forced to do it.”

After learning of the administration’s decision, Mr. Blumenauer’s office celebrated “a quiet victory,” but urged supporters not to crow about it.

“While we are very happy with the result, we won’t be shouting it from the rooftops because we aren’t out of the woods yet,” Mr. Blumenauer’s office said in an e-mail in early November to people working with him on the issue. “This regulation could be modified or reversed, especially if Republican leaders try to use this small provision to perpetuate the ‘death panel’ myth.”

Moreover, the e-mail said: “We would ask that you not broadcast this accomplishment out to any of your lists, even if they are ‘supporters’ — e-mails can too easily be forwarded.”

The e-mail continued: “Thus far, it seems that no press or blogs have discovered it, but we will be keeping a close watch and may be calling on you if we need a rapid, targeted response. The longer this goes unnoticed, the better our chances of keeping it.”

In the interview, Mr. Blumenauer said, “Lies can go viral if people use them for political purposes.”

The proposal for Medicare coverage of advance care planning was omitted from the final health care bill because of the uproar over unsubstantiated claims that it would encourage euthanasia.

Sarah Palin, the 2008 Republican vice-presidential candidate, and Representative John A. Boehner of Ohio, the House Republican leader, led the criticism in the summer of 2009. Ms. Palin said “Obama’s death panel” would decide who was worthy of health care. Mr. Boehner, who is in line to become speaker, said, “This provision may start us down a treacherous path toward government-encouraged euthanasia.” Forced onto the defensive, Mr. Obama said that nothing in the bill would “pull the plug on grandma.”

A recent poll by the Kaiser Family Foundation suggests that the idea of death panels persists. In the September poll, 30 percent of Americans 65 and older said the new health care law allowed a government panel to make decisions about end-of-life care for people on Medicare. The law has no such provision.

The new policy is included in a huge Medicare regulation setting payment rates for thousands of services including arthroscopy, mastectomy and X-rays.

The rule was issued by Dr. Donald M. Berwick, administrator of the Centers for Medicare and Medicaid Services and a longtime advocate for better end-of-life care.

“Using unwanted procedures in terminal illness is a form of assault,” Dr. Berwick has said. “In economic terms, it is waste. Several techniques, including advance directives and involvement of patients and families in decision-making, have been shown to reduce inappropriate care at the end of life, leading to both lower cost and more humane care.”

Ellen B. Griffith, a spokeswoman for the Medicare agency, said, “The final health care reform law has no provision for voluntary advance care planning.” But Ms. Griffith added, under the new rule, such planning “may be included as an element in both the first and subsequent annual wellness visits, providing an opportunity to periodically review and update the beneficiary’s wishes and preferences for his or her medical care.”

Mr. Blumenauer and Mr. Rockefeller said that advance directives would help doctors and nurses provide care in keeping with patients’ wishes.

“Early advance care planning is important because a person’s ability to make decisions may diminish over time, and he or she may suddenly lose the capability to participate in health care decisions,” the lawmakers said in a letter to Dr. Berwick in August.

In a recent study of 3,700 people near the end of life, Dr. Maria J. Silveira of the University of Michigan found that many had “treatable, life-threatening conditions” but lacked decision-making capacity in their final days. With the new Medicare coverage, doctors can learn a patient’s wishes before a crisis occurs.

For example, Dr. Silveira said, she might ask a person with heart disease, “If you have another heart attack and your heart stops beating, would you want us to try to restart it?” A patient dying of emphysema might be asked, “Do you want to go on a breathing machine for the rest of your life?” And, she said, a patient with incurable cancer might be asked, “When the time comes, do you want us to use technology to try and delay your death?”

    Obama Returns to End-of-Life Plan That Caused Stir, NYT, 25.12.2010, http://www.nytimes.com/2010/12/26/us/politics/26death.html

Avastin

December 25, 2010
The New York Times

The Food and Drug Administration is preparing to withdraw its approval for using Avastin, a tumor-slowing drug, to treat advanced breast cancers that have spread to other parts of the body. It was a reasonable decision, based on scientific evidence. The drug has failed to extend lives in clinical trials, and it carries a small risk of devastating, sometimes fatal, side effects.

The agency’s move has brought protests from some patients who think Avastin is helping them and charges from conservative critics that this is a step toward rationing. If the F.D.A. does rescind its approval, insurers may stop covering the drug for breast cancer. Few patients can afford to pay the exorbitant costs — as high as $88,000 a year — out of pocket.

We believe the F.D.A. has shown courage in following the scientific evidence on this highly emotional issue. Even some advocacy groups for breast cancer patients have applauded the agency’s decision for making clear that the drug does not work very well.

Women whose breast cancer has spread have few good options. Their advanced disease is essentially incurable, so the best they can hope for is a treatment that will extend their lives or improve their quality of life.

Two years ago the F.D.A., bending over backward to be compassionate, gave Avastin “accelerated approval” for breast cancer treatment based on a single, unimpressive clinical trial. The results showed that Avastin, when added to standard chemotherapy, slowed the progression of the tumors for 5.5 months for the median user but did not extend lives. Two follow-up trials submitted by the manufacturer, Genentech, found tumor progression was held at bay for even shorter periods, from one to three months, and again, lives were not extended.

Worse yet, some patients suffered serious, disabling side effects, including severe bleeding, stroke or heart problems, severe high blood pressure and development of holes in the gastrointestinal tract. Roughly 1 percent of the patients died from Avastin-related causes.

Expert opinion is split on whether the extremely modest benefits of the drug are worth the risks. An advisory committee to the F.D.A. voted 12 to 1 in July that the approval for breast cancer should be withdrawn. The top British advisory group declined this month to recommend the drug for breast cancer because of its uncertain benefits and lack of proof it extended survival. However, the top advisory agency for the European Union ruled this month that the benefits outweighed the risks.

Genentech plans to request a hearing with the F.D.A. to argue the case for retaining Avastin’s status as an approved breast cancer treatment. It should focus on proposing ways to identify the subset of women who can really benefit from Avastin.

    Avastin, NYT, 25.12.2010, http://www.nytimes.com/2010/12/26/opinion/26sun2.html

Hospitals Send Bill After Mental Patients Pursue Damages

December 24, 2010
The New York Times
By ALISON LEIGH COWAN

Daniel J. Langevin was 35, mentally ill and broke. He had been living in psychiatric institutions on and off since his early 20s.

A friend who visited him at the Rochester Psychiatric Center in February 1995 remembered that Mr. Langevin had pain in his jaw, eye and face that was not getting much attention from the staff. A week later, he was discovered unconscious, with a near-fatal infection spreading to his brain and other organs.

Mr. Langevin sued New York State, which operates the hospital, and probably would have won a sizable award. But the state countered by demanding that Mr. Langevin reimburse it $1.7 million for 10 years of inpatient care he had received. A judge sided with the state, and Mr. Langevin wound up with nothing.

Slip and fall in a New York prison, or suffer abuse by its guards, and inmates can keep whatever they win in court. But for patients in state-run mental hospitals — people too ill to live on their own and too poor to pay for their care — the state can drain court-awarded damages, effectively deducting the cost of their stays in the very hospitals that failed or abused them.

“It’s a Catch-22, isn’t it?” said Leo G. Finucane, the lawyer who represented Mr. Langevin. “I need to go to this facility because I’m sick. But if they hurt me worse, they’re immune.”

Attorney General Andrew M. Cuomo’s office, which handles these kinds of cases for New York State, declined repeated requests to discuss the matter.

It is not uncommon for public hospitals to lean hard on patients found to have assets. But New York squeezes patients who sue for injuries more consistently and harder than many other states, according to lawyers and others who represent the mentally ill.

“I’ve done this work since 1986, and New York has had the reputation of doing this far more than any other state in the country,” Susan Stefan, a lawyer and professor, said.

Lawyers in Oregon still talk about the time their state forced a patient to hand over 80 percent of the settlement he received from the Roman Catholic Church as a victim of sexual abuse. But in 1999, Oregon amended its law to keep the state from satisfying hospital debts with money that patients obtained from the state itself for acts of negligence.

In New Jersey, the state five years ago stopped imposing liens on psychiatric patients with unpaid balances, and lawyers for injured or abused patients can have clauses inserted into judgments and settlements promising that the state will not go after the awards to satisfy hospital debts, Joseph Young, executive director of the advocacy group Disability Rights New Jersey, said.

In Wisconsin, the state sought a $1,000-per-night reimbursement for the years an indigent woman stayed at the Mendota Mental Health Institute, after the woman choked to death on a peanut butter sandwich she should not have been given and her family filed a wrongful-death suit. But on the eve of trial, the family’s lawyer negotiated a deal that left her heirs with thousands of dollars in compensation and a promise that the state would waive its claim. “Even the state recognizes that what the law allows it to do may result in extreme and unjust consequences,” the lawyer, Jason Studinski, said.

Over the years, New York has exercised its right to collect in dozens of these kinds of cases and has generally received a go-ahead from the courts.

There was the 20-year-old woman raped at South Beach Psychiatric Center on Staten Island in 1992. Six years later, a judge deducted $101,000 from her $250,000 award to cover what the state contended she owed for services; the judge ruled that a hospital might be negligent on some days while providing valuable services on others. The patient appealed, to no avail.

Ron Lynch, a patient at Bronx Psychiatric Center, sued for $7 million in damages after being assaulted in 2003 by another patient. This prompted the state to respond that Mr. Lynch ought to pay $1,585,519.22 to reduce the bill he had run up for his treatment.

Evelyn Hasson was given a toxic amount of the antipsychotic drug Thorazine at Manhattan Psychiatric Center in 1993. Her relatives brought a wrongful-death suit, and the state sent a bill of $220,136.90 to her estate. The parties reached a financial settlement that will net her family $100,000, according to her lawyer, Bill Brooks of the Touro Law Center, but the questions of whether the counterclaim violated her constitutional rights have been pending in federal court for 11 years.

The state courts in New York have generally allowed the state to pursue reimbursement, but with limits, because of a patient named Louis O. Acevedo.

Mr. Acevedo sued the state after breaking his leg during an assault at Middletown Psychiatric Center in 1986, and he was soon warned that he could face a bill for $265,647.66. He withdrew his claim but challenged in federal court the state’s use of counterclaims. A judge ruled in 1991 that the state should be seeking reimbursement only up to the amount a patient recovered. Anything larger, the judge said, would deter patients from seeking redress because of the possibility that they might end up worse off.

In the years since then, patients’ lawyers have argued that even with a cap, letting the state claw back its own negligence awards is a disincentive to sue.

In the Langevin case, Judge Donald J. Corbett Jr. of the New York Court of Claims, like other jurists who have examined the issue, found in favor of the state. New York’s mental hygiene law gives the state the right to go after found assets or windfalls and allows six years from when the debt was incurred to begin an action against a patient.

“That does smack me as being unfair, especially in a small case,” said G. Oliver Koppell, a city councilman from the Bronx who temporarily served as New York’s attorney general in 1994. “If someone recovers $1 million and has a hospital bill of $50,000, it doesn’t bother me because they’re now a wealthy person. But for someone who has no money, to have it all taken back, it does strike me as being unfair or punitive.”

Maria Randone, 52, who has been in and of New York institutions since she was 14, is testing the law anew.

Ms. Randone was at an assisted-living home in Highland, N.Y., in 2005, but after smashing her plate in the dining room and causing a scene, she was taken away by the police and was involuntarily committed to a nearby hospital.

“I got a little upset because I wanted seconds,” Ms. Randone later testified.

Hudson River Psychiatric Center, where she was placed, let her shower soon after without assistance, despite her documented need for help. Staff members found her sprawled at the bottom of the stall, with a broken hip and femur.

Ms. Randone sued in 2007, and the state parried with a countersuit preserving its right to have any windfall garnished to repay the $467,051.94 bill it calculated she owed for two years of her stay in state-run psychiatric hospitals.

Last January, the Court of Claims in White Plains found the state fully responsible for Ms. Randone’s fall, entitling her to damages for pain, suffering and impairment. The court examined how much to award her — and how much should go back to the state — last month in a second trial. A ruling is expected by April.

Richard Greenblatt, the lawyer in Poughkeepsie who is representing Ms. Randone, said he thought the injury would ordinarily justify damages of $400,000 to $1 million. He said that he had tried to settle the case for $500,000, of which $100,000 would be returned to the state to cover the hospital bill, but that the assistant attorney general currently on the case showed no interest.

He “rejected the proposal outright, saying if she has the money, we’re going to take it,” Mr. Greenblatt said, adding: “It just reeks of unfairness. It’s Kafkaesque.”

Mr. Finucane, Mr. Langevin’s lawyer, struggles to see how the approach is good for hospitals or patients. He said state employees “don’t have to be careful if the state has no liability and if there’s no pressure from above to be careful on the floors because ‘we don’t want to be shelling out millions of dollars for negligence claims.’

“They’ve got this wonderful way of addressing it,” Mr. Finucane continued. “ ‘Well, we’ll just ask them to pay for everything we’ve done for them for several years.’ ”

    Hospitals Send Bill After Mental Patients Pursue Damages, NYT, 25.12.2010, http://www.nytimes.com/2010/12/25/nyregion/25damages.html

Many Children Lack Doctors, Study Finds

December 19, 2010
The New York Times
By THE ASSOCIATED PRESS

CHICAGO (AP) — Though there are enough children’s doctors in the United States, they work in the wrong places, a new study has found.

Some wealthy areas are oversaturated with pediatricians and family doctors, while other parts of the nation have few or none.

Nearly 1 million children live in areas with no local doctor. By relocating doctors, the study suggests, nearly every child could have one nearby.

The study’s lead author, Dr. Scott Shipman of the Dartmouth Institute for Health Policy and Clinical Practice, said the focus should be more on evening out the distribution of doctors than on increasing the supply.

Medical schools are graduating more students, Dr. Shipman said, but the result will most likely be more doctors in places where there is already an oversupply.

“I worry that it could get worse,” he said.

The growth in the number of pediatricians and family physicians has outpaced the growth in the child population in the United States, Dr. Shipman and his colleagues found. Yet the study’s analysis shows that nearly all 50 states have an extremely uneven distribution of primary care doctors for children.

Mississippi had the highest proportion of children, 42 percent, in low-supply regions, which are defined as areas with more than 3,000 children per doctor. Next were Arkansas, Oklahoma, Maine and Idaho.

Areas with an abundance of children’s doctors were Washington, D.C., and Delaware, both of which had no children living in low-supply regions. Maryland, Washington State and Wisconsin also had very few children in low-supply areas.

Federal financing has been expanded in recent years for the National Health Service Corps, which offers loan forgiveness for doctors and other health practitioners who work in underserved areas. That may help, Dr. Shipman said.

The study appears Monday in the journal Pediatrics.

    Many Children Lack Doctors, Study Finds, NYT, 19.12.2010, http://www.nytimes.com/2010/12/20/us/20doctors.html

Serious Mental Health Needs Seen Growing at Colleges

December 19, 2010
The New York Times
By TRIP GABRIEL

STONY BROOK, N.Y. — Rushing a student to a psychiatric emergency room is never routine, but when Stony Brook University logged three trips in three days, it did not surprise Jenny Hwang, the director of counseling.

It was deep into the fall semester, a time of mounting stress with finals looming and the holiday break not far off, an anxiety all its own.

On a Thursday afternoon, a freshman who had been scraping bottom academically posted thoughts about suicide on Facebook. If I were gone, he wrote, would anybody notice? An alarmed student told staff members in the dorm, who called Dr. Hwang after hours, who contacted the campus police. Officers escorted the student to the county psychiatric hospital.

There were two more runs over that weekend, including one late Saturday night when a student grew concerned that a friend with a prescription for Xanax, the anti-anxiety drug, had swallowed a fistful.

On Sunday, a supervisor of residence halls, Gina Vanacore, sent a BlackBerry update to Dr. Hwang, who has championed programs to train students and staff members to intervene to prevent suicide.

“If you weren’t so good at getting this bystander stuff out there,” Ms. Vanacore wrote in mock exasperation, “we could sleep on the weekends.”

Stony Brook is typical of American colleges and universities these days, where national surveys show that nearly half of the students who visit counseling centers are coping with serious mental illness, more than double the rate a decade ago. More students take psychiatric medication, and there are more emergencies requiring immediate action.

“It’s so different from how people might stereotype the concept of college counseling, or back in the ’70s students coming in with existential crises: who am I?” said Dr. Hwang, whose staff of 29 includes psychiatrists, clinical psychologists and social workers. “Now they’re bringing in life stories involving extensive trauma, a history of serious mental illness, eating disorders, self-injury, alcohol and other drug use.”

Experts say the trend is partly linked to effective psychotropic drugs (Wellbutrin for depression, Adderall for attention disorder, Abilify for bipolar disorder) that have allowed students to attend college who otherwise might not have functioned in a campus setting.

There is also greater awareness of traumas scarcely recognized a generation ago and a willingness to seek help for those problems, including bulimia, self-cutting and childhood sexual abuse.

The need to help this troubled population has forced campus mental health centers — whose staffs, on average, have not grown in proportion to student enrollment in 15 years — to take extraordinary measures to make do. Some have hospital-style triage units to rank the acuity of students who cross their thresholds. Others have waiting lists for treatment — sometimes weeks long — and limit the number of therapy sessions.

Some centers have time only to “treat students for a crisis, bandaging them up and sending them out,” said Denise Hayes, the president of the Association for University and College Counseling Center Directors and the director of counseling at the Claremont Colleges in California.

“It’s very stressful for the counselors,” she said. “It doesn’t feel like why you got into college counseling.”

A recent survey by the American College Counseling Association found that a majority of students seek help for normal post-adolescent trouble like romantic heartbreak and identity crises. But 44 percent in counseling have severe psychological disorders, up from 16 percent in 2000, and 24 percent are on psychiatric medication, up from 17 percent a decade ago.

The most common disorders today: depression, anxiety, suicidal thoughts, alcohol abuse, attention disorders, self-injury and eating disorders.

Stony Brook, an academically demanding branch of the State University of New York (its admission rate is 40 percent), faces the mental health challenges typical of a big public university. It has 9,500 resident students and 15,000 who commute from off-campus. The highly diverse student body includes many who are the first in their families to attend college and carry intense pressure to succeed, often in engineering or the sciences. A Black Women and Trauma therapy group last semester included participants from Africa, suffering post-traumatic stress disorder from violence in their youth.

Stony Brook has seen a sharp increase in demand for counseling — 1,311 students began treatment during the past academic year, a rise of 21 percent from a year earlier. At the same time, budget pressures from New York State have forced a 15 percent cut in mental health services over three years.

Dr. Hwang, a clinical psychologist who became director in July 2009, has dealt with the squeeze by limiting counseling sessions to 10 per student and referring some, especially those needing long-term treatment for eating disorders or schizophrenia, to off-campus providers.

But she has resisted the pressure to offer only referrals. By managing counselors’ workloads, the center can accept as many as 60 new clients a week in peak demand between October and the winter break.

“By this point in the semester to not lose hope or get jaded about the work, it can be a challenge,” Dr. Hwang said. “By the end of the day, I go home so adrenalized that even though I’m exhausted it will take me hours to fall asleep.”

For relief, she plays with her 2-year-old daughter, and she has taken up the guitar again.

Shifting to Triage

Near the student union in the heart of campus, the Student Health Center building dates from the days when a serious undergraduate health problem was mononucleosis. But the hiring of Judy Esposito, a social worker with experience counseling Sept. 11 widows, to start a triage unit three years ago was a sign of the new reality in student mental health.

At 9 a.m. on the Tuesday after the campus’s very busy weekend, Ms. Esposito had just passed the Purell dispenser by the entrance when she noticed two colleagues hurrying toward her office. Before she had taken off her coat, they were updating her about a junior who had come in the previous week after cutting herself and expressing suicidal thoughts.

Ms. Esposito’s triage team fields 15 to 20 requests for help a day. After brief interviews, most students are scheduled for a longer appointment with a psychologist, which leads to individual treatment. The one in six who do not become patients are referred to other university departments like academic advising, or to off-campus therapists if long-term help is needed. There are no charges for on-campus counseling.

This day the walk-ins included a young man complaining of feeling friendless and depressed. Another student said he was struggling academically, feared that his parents would find out and was drinking and feeling hopeless.

Professionals in a mental health center are mindful of their own well-being. For this reason the staff had planned a potluck holiday lunch. While a turkey roasted in the kitchen that serves as the break room, Ms. Esposito helped warm up candied yams, stuffing and the store-bought quiche that was her own contribution.

Just then Regina Frontino, the triage assistant who greets walk-ins at the front desk, swept into the kitchen to say a student had been led in by a friend who feared that she was suicidal.

Ms. Esposito rushed to the lobby. From a brief conversation, she knew that the distraught student would have to go to the hospital. The counseling center does not have the ability to admit suicidal or psychotic students overnight for observation or to administer powerful drugs to calm them. It arranges for them to be taken to the Stony Brook University Medical Center, on the far side of the 1,000-acre campus. The hospital has a 24-hour psychiatric emergency room that serves all of Suffolk County.

“They’re not going to fix what’s going on,” Ms. Esposito said, “but in that moment we can ensure she’s safe.” She called Tracy Thomas, an on-call counselor, to calm the student, who was crying intermittently, while she phoned the emergency room and informed Dr. Hwang, who called the campus police to transport the young woman.

When Ms. Esposito heard the crackle of police radios in the hallway, she went to tell the student for the first time that she would have to go to the hospital.

“This is not something students love to do,” Ms. Esposito recounted. The young woman told her she did not want to go. Ms. Esposito replied that the staff was worried for her safety, and she repeated the conversation she had had earlier with the young woman:

Are you having thoughts about wanting to die?

Yes.

Are you afraid you are actually going to kill yourself?

Yes.

She invited a police officer into the counseling room, and the student teared up again at the sight of him. Ms. Esposito assured her that she was not in trouble. Meanwhile, an ambulance crew arrived with a rolling stretcher, but the young woman walked out on her own with the officers.

Because Ms. Thomas, a predoctoral intern in psychology, now needed to regain her own equilibrium before seeing other clients, Ms. Esposito debriefed her about what had just happened.

Finally she returned to her office, having missed the holiday lunch, and found that her team had prepared a plate for her.

“It’s kind of like firemen,” she said. “When the fire’s on, we are just at it. But once the fire’s out, we can go back to the house and eat together and laugh.”

Reaching Out

Even though the appointment books of Stony Brook counselors are filled, all national evidence suggests that vastly more students need mental health services.

Forty-six percent of college students said they felt “things were hopeless” at least once in the previous 12 months, and nearly a third had been so depressed that it was difficult to function, according to a 2009 survey by the American College Health Association.

Then there is this: Of 133 student suicides reported in the American College Counseling Association’s survey of 320 institutions last year, fewer than 20 had sought help on campus.

Alexandria Imperato, 23, remembers that as a Stony Brook freshman all her high school friends were talking about how great a time they were having in college, while she felt miserable. She faced family issues and the pressure of adjusting to college. “You go home to Thanksgiving dinner, and the family asks your brother how is his gerbil, and they ask you, ‘What are doing with the rest of your life?’ ” Ms. Imperato said.

She learned she had clinical depression. She eventually conquered it with psychotherapy, Cymbalta and lithium. She went on to form a Stony Brook chapter of Active Minds, a national campus-based suicide-prevention group.

“I knew how much better it made me feel to find others,” said Ms. Imperato, who plans to be a nurse.

On recent day, she was one of two dozen volunteers in black T-shirts reading “Chill” who stopped passers-by in the Student Activities Center during lunch hour.

“Would you like to take a depression screening?” they asked, offering a clipboard with a one-page form to all who unplugged their ear buds. Students checked boxes if they had difficulty sleeping, felt hopeless or “had feelings of worthlessness.” They were offered a chance to speak privately with a psychologist in a nearby office. Sixteen said yes.

The depression screenings are part of a program to enlist students to monitor the mental health of peers, which is run by the four-year-old Center for Outreach and Prevention, a division of mental health services that Dr. Hwang oversaw before her promotion to director of all counseling services.

She is committed to outreach in its many forms, including educating dormitory staff members to recognize students in distress and encouraging professors to report disruptive behavior in class.

In previous years, more than 1,000 depression screenings were given to students, with 22 percent indicating signs of major depression. Dr. Hwang credits that and other outreach efforts to the swell of new cases for counseling. “For a lot of people it’s terrifying” to come to the counseling center, she said. “If there’s anything we can do to make it easier to walk in, I feel like we owe it to them.”

Stony Brook has not had a student suicide since spring 2009, unusual for a campus its size. But Dr. Hwang is haunted by the impact on the campus of several off-campus student deaths in accidents and a homicide in the past year. “With every vigil, with every conversation with someone in pain, there’s this overwhelming sense of we need to learn something,” she said. “I think about these parents who’ve invested so much into getting their kids alive to 18.”

One student who said yes to an impromptu interview with a counselor after filling out a depression screening was a psychology major, a senior from upstate New York. As it happened, Dr. Hwang had wandered over from the counseling center to check on the screenings, and the young woman spent a long time conferring with her, never removing her checked coat or backpack.

“I don’t have motivation for things anymore,” the student said afterward. “This place just depresses me the whole time.”

She had been unaware that students could walk in unannounced to the counseling center. “I thought you had to make an appointment,” she said. “Yes,” she said, “I’ll do that.”

    Serious Mental Health Needs Seen Growing at Colleges, NYT, 19.12.2010, http://www.nytimes.com/2010/12/20/health/20campus.html

Transplants Cut, Arizona Is Challenged by Survivors

December 18, 2010
The New York Times
By MARC LACEY

PHOENIX — First, it was distraught patients awaiting organ transplants who protested Arizona’s decision to no longer cover such operations under its Medicaid program.

Now, Arizonans who received such transplants, and are alive and well as a result of them, are questioning the data that lawmakers relied on to make their controversial benefit cuts.

“They say it’s too expensive,” said Star Boelter, 52, who had a stem cell transplant that was paid for by Arizona’s Medicaid program in 2009 after suffering from leukemia. “Well, how much is life worth? They say most people die. Well, I’m alive because of my transplant.”

When Arizona lawmakers voted last spring to cut some state-financed transplant coverage, they relied on data provided by state health officials showing that the procedures were rarely successful. But transplant experts and some patients who have undergone the now-discontinued procedures question the state’s numbers.

For bone marrow transplants, the legislators were told that 13 of 14 patients covered by the state’s Medicaid program who underwent that procedure died within six months. The 14th patient could not be tracked, state health officials told the Legislature, and might have died as well.

But Kim Marie Urick, a leukemia survivor, wants the state’s leaders to know that she is able to ride her three horses outside Sedona and spend time with her husband and son thanks to a bone marrow transplant that Arizona’s Medicaid program paid for on June 4, 2009.

“I was about five days away from dying,” she said in a telephone interview. “I essentially had no immune system. If it wasn’t for the bone marrow transplant, I wouldn’t be here right now.”

The cure rate for bone marrow transplants cited in the report to the Legislature was either zero or 7 percent, depending on whether that unidentified 14th patient lived. But transplant experts put the actual survival rate, based on national studies, at over 40 percent.

Dr. Jeffrey R. Schriber, medical director of the Blood and Marrow Transplant Program at Banner Good Samaritan Medical Center in Phoenix, has written to Gov. Jan Brewer and state lawmakers telling them that their decisions were based on incomplete data that gave the wrong picture. His data show the success rate for bone marrow transplants covered by Arizona’s Medicaid program at slightly higher than the national average. Of 20 operations performed at Banner in recent years, 9 patients have survived, he said.

State Representative John Kavanagh, a Republican and chairman of the House Appropriations Committee, has said that if new data was presented, he would be willing to reconsider at least some of the cuts to the Arizona Health Care Containment System, as the Medicaid program is known, when the Legislature returns to session next month to tackle the state’s budget crisis.

But Ms. Brewer has not acknowledged that the cuts she signed into law might have been based on incomplete data. She repeated the figure that 13 out of 14 bone marrow transplant recipients died in Arizona while discussing the issue with Greta van Susteren on Fox News last week. “Because Arizona has been hit with a devastating budget deficit, we have had to do some difficult decisions,” she said, adding later, “We have no other choices.”

But Democrats disagree, especially State Representative Anna Tovar, who underwent a bone marrow transplant herself a decade ago, although not as part of the state Medicaid plan. She has been among the most vocal critics of the transplant cuts, calling for a special session this month to reconsider the decision.

Ms. Tovar’s body rejected the bone marrow transplant in 2001, and she then underwent a stem cell transplant the next year. “I’m living proof that these transplants do work,” she said.

Ms. Urick, 53, still remembers when she learned that she was being considered for a transplant that might extend her life. “To be told there’s a way you can live is one of the most wonderful things you can ever hear,” she said. “I can’t imagine what those who are waiting for transplants now and can’t afford them are going through.”

As for her, Ms. Urick said, “I plan on living another 20, 30 or 40 years.”

Ms. Boelter, a massage therapist, is back to providing relief to others. “I’m working,” she said. “I’m paying taxes, just as I’ve done for most of my life.”

Another leukemia survivor, Michael Cheshaek, 27, who underwent a bone marrow transplant in 2008, remains on disability but still credits the operation with allowing him to live.

“We send money all over the world to help people, and those who are suffering at home are not getting the help they need,” said Mr. Cheshaek, whose operation was covered under his private insurance coverage, which his mother supplemented with money from her retirement plan.

Bone marrow transplants are not the only ones in which legislators used questionable data to make their decision, transplant experts say. The American Society of Transplant Surgeons called Arizona’s transplant cuts “decisions with no medical justification.”

Liver transplants for those with hepatitis C, which the state also discontinued, have a survival rate exceeding 80 percent after one year and 60 percent after five years, the transplant group said. Arizona’s study of such procedures was far more pessimistic, saying such transplants “do not significantly affect the diseases they are intended to cure.”

For lung transplants, the transplant society called them “life saving, not palliative,” in response to the Arizona study, which labeled them “more palliative than curative.”

James Healy, 25, a student at Arizona State University, had a state-financed bone marrow transplant in 2009 and is back at school part time studying applied psychology. “I’m well on my way to recovery,” said Mr. Healy, who suffered from leukemia. “I’ve started school again, and I’m getting out and about. I’ve seen other people go through it, and I’ve gone through it. We’re very much alive.”

    Transplants Cut, Arizona Is Challenged by Survivors, NYT, 18.12.2010, http://www.nytimes.com/2010/12/19/us/19transplant.html

Early Tests for Alzheimer’s Pose Diagnosis Dilemma

December 17, 2010
The New York Times
By GINA KOLATA

Marjie Popkin thought she had chemo brain, that fuzzy-headed forgetful state that she figured was a result of her treatment for ovarian cancer. She was not thinking clearly — having trouble with numbers, forgetting things she had just heard.

One doctor after another dismissed her complaints. Until recently, since she was, at age 62, functioning well and having no trouble taking care of herself, that might have been the end of her quest for an explanation.

Last year, though, Ms. Popkin, still troubled by what was happening to her mind, went to Dr. Michael Rafii, a neurologist at the University of California, San Diego, who not only gave her a thorough neurological examination but administered new tests, like an M.R.I. that assesses the volume of key brain areas and a spinal tap.

Then he told her there was something wrong. And it was not chemo brain. It most likely was Alzheimer’s disease. Although she seemed to be in the very early stages, all the indicators pointed in that direction.

Until recently, the image of Alzheimer’s was the clearly demented person with the sometimes vacant stare, unable to follow a conversation or remember a promise to meet a friend for lunch.

Ms. Popkin is nothing like that. To a casual observer, the articulate and groomed Ms. Popkin seems perfectly fine. She is in the vanguard of a new generation of Alzheimer’s patients, given a diagnosis after tests found signs of the disease years before actual dementia sets in.

But the new diagnostic tests are leading to a moral dilemma. Since there is no treatment for Alzheimer’s, is it a good thing to tell people, years earlier, that they have this progressive degenerative brain disease or have a good chance of getting it?

“I am grappling with that issue,” Dr. Rafii said. “I give them the diagnosis — we are getting pretty good at diagnosis now. But it’s challenging because what do we do then?”

It is a quandary that is emblematic of major changes in the practice of medicine, affecting not just Alzheimer’s patients. Modern medicine has produced new diagnostic tools, from scanners to genetic tests, that can find diseases or predict disease risk decades before people would notice any symptoms.

At the same time, many of those diseases have no effective treatments. Does it help to know you are likely to get a disease if there is nothing you can do?

“This is the price we pay” for the new knowledge, said Dr. Jonathan D. Moreno, a professor of medical ethics and the history and sociology of science at the University of Pennsylvania.

“I think we are going to go through a really tough time,” he added. “We have so much information now, and we have to try to learn as a culture what information we do not want to have.”

Some doctors, like Dr. John C. Morris of Washington University in St. Louis, say they will not offer the new diagnostic tests for Alzheimer’s — like M.R.I.’s and spinal taps — to patients because it is not yet clear how to interpret them. He uses them in research studies but does not tell subjects the results.

“We don’t know for certain what these results mean,” Dr. Morris said. “If you have amyloid in your brain, we don’t know for certain that you will become demented, and we don’t have anything we can do about it.”

But many people want to know anyway and say they can handle the uncertainty.

That issue is facing investigators in a large federal study of early signs of Alzheimer’s. The researchers, who include Dr. Morris, have been testing and following hundreds of people aged 55 to 90, some with normal memories, some with memory problems and some with dementia. So far, only investigators know the results. Now, the question is, should those who want to learn what their tests show be told?

“We are just confronting this,” said Dr. Richard J. Hodes, director of the National Institute on Aging. “Bioethicists are talking with scientists and the public about what is the right thing to do.”

Risk Levels, but No Scores

Dr. Rafii learned about the new tests and how to use them because he is an investigator in that large federal study. But many who come to the memory disorders clinic at the University of California, San Diego, where Dr. Rafii works, are not part of that study, the Alzheimer’s Disease Neuroimaging Initiative, and simply want to know what is wrong with their brains.

So Dr. Rafii sometimes offers the study’s diagnostic tests: spinal taps and M.R.I.’s to look for shrinkage in important areas of the brain; PET scans to look for the telltale signs of Alzheimer’s in the brain. He calls it “ADNI in the real world,” referring to the study’s acronym. Others, too, offer such tests, although doctors differ in how far they will go.

Dr. Mony J. de Leon of New York University, for example, takes a middle ground. He is studying people at increased risk for Alzheimer’s or other dementias, especially those whose mothers had Alzheimer’s. That sort of family history, he has found, makes the disease more likely.

Many who come to his clinic have no memory problems, but are worried. So Dr. de Leon enrolls them in a study and regularly subjects them to an array of tests — ones that probe their memory, and ones like spinal taps and brain scans that look for signs of Alzheimer’s. But he only provides people with a sort of general assessment, telling them they are at increased risk, decreased risk or somewhere in the middle.

“We do not reveal their scores,” Dr. de Leon said.

Some are satisfied with that.

Enzo Simone, for example, learned that, at age 43, his tests results do not indicate an increased risk. He was glad to know, but is not convinced he has escaped what he sees as a family fate. His mother, grandmother and great-grandmother had Alzheimer’s. They got the disease in their 60s. Mr. Simone, who lives in Amawalk, N.Y., reasoned that he had 20 years before it was likely to strike. And he intends to continue being tested as part of Dr. de Leon’s study.

And for those who demand more details than just a “yes,” “no” or “maybe,” Dr. de Leon refuses.

“We say, ‘It is a statistical exercise, it is a proof of concept, it is a baby test,’ ” Dr. de Leon said. “Some say: ‘That’s not good enough. I come back to you every year, and if you want me to continue I need more than that.’ ”

But Dr. de Leon said he was constrained by his hospital’s ethics board, which has to approve his studies. It is extremely difficult, he says, to convince the board that giving out uncertain information about risk can help people, given that there is no effective treatment. And so, he says, he tells patients, “You are here to do an experiment.”

Others, like Dr. Lawrence Honig of Columbia University, say they sometimes see patients with no symptoms of memory loss who are nonetheless worried about their risk. Some of them have already gotten one of the early diagnostic tests, like a spinal tap or brain scan, from a neurologist in private practice, and have been told they were on their way to developing Alzheimer’s. They come to Dr. Honig, hoping he will say it is not true.

That situation, Dr. Honig says, “has become more and more common over the last few years.” He says that when test results are consistent with Alzheimer’s, he is honest about it, telling patients that the results are “suggestive of Alzheimer’s” but adding that all he can say for sure is “at some point in the future, you might be faced with that condition.”

He agonizes, though, over telling people news like that.

“I think it’s pretty terrible,” Dr. Honig said. “It is psychologically invasive.”

But for neurologists like Dr. de Leon, the future is fast approaching, as patients increasingly demand to know.

“The floodgate is about to open,” Dr. de Leon said.

Information’s Burden

At Boston University, Dr. Robert Green faced an ethical dilemma. He wanted to test people for a gene, APOE, that has three variants. People with two copies of one of the variants, APO e4, have a 12- to 15-fold increased risk of Alzheimer’s disease. People with even one copy of the gene variant have about a threefold increased risk.

Five different published consensus statements by ethicists and neurologists had considered the question of whether people should be told the results of APOE tests. And every one of those committee said the answer is no, do not tell.

Dr. Green wondered if that answer was right.

“It seemed rather strange to be in a position where family members are coming to you and saying, ‘I really understand APOE genotyping and the idea of a risk gene, and I want to know my genotype,’ and then to say to them, ‘I could tell you that, but I’m not going to.’ ” After all, he said, “Part of what we do in medicine is to inform.”

He knew what it meant to tell people they were at high risk.

“Alzheimer’s is a fearsome disease,” he said. “You can’t get much more fearsome than Alzheimer’s.” And yet, he said, “People still wanted to know.”

He decided to do a study to see what would happen if he told.

The first surprise was how many people wanted to know. To be in the study, a person had to have a first-degree relative who had had Alzheimer’s, making it more likely that they would have an APO e4 variant. Dr. Green thought maybe a small percentage of the people he approached would want to have the genetic test. Instead, nearly a quarter did.

“Frankly, we were terrified in early days of this study,” Dr. Green said. “We did not want to harm anyone. We were very, very thoughtful and intense. We sat with people beforehand and asked if they were really sure they wanted to do this.”

But his subjects were fine with the testing. After they gave the subjects their test results, researchers looked for psychological effects, observing participants in conversations and administering standardized questions designed to detect anxiety or depression or suicidal thoughts. They found nothing.

The main difference between those who found out they had APO e4 and those who found out they did not have that gene variant is that the APO e4 subjects were more likely to buy long term care insurance, were more likely to start exercising and were more likely to start taking vitamins and nutritional supplements, even though these practices and products have never been shown to protect against Alzheimer’s.

For many, though, the news was good — they did not have APO e4.

That is what happened with Alan Whitney, a 66-year-old radio astronomer at the Massachusetts Institute of Technology, whose mother and mother’s father both had Alzheimer’s. He wanted the test, he said, knowing what it meant to have APO e4.

“That was a gamble I took,” he said.

And he was lucky. If he had had an APO e4 gene, he said, he might have taken early retirement and traveled.

“Now I feel I have some time.” Dr. Whitney said.

Robert Stuart-Vail, an 83-year-old retired columnist for his local newspaper in Lincoln, Mass., was not so lucky. He found out he has one copy of the APO e4 gene. His father had Alzheimer’s and so did his wife, who died from it, so he knows full well what the disease entails

“I wanted to know,” Mr. Stuart-Vail said. “I wanted to be able to tell my children.”

When he told them, though, they did not say much, Mr. Stuart-Vail said.

“I don’t think it meant that much to them. Alzheimer’s — that’s something that happens to old people.” Mr. Stuart-Vail’s children are middle-aged.

As for Mr. Stuart-Vail, he believes staying active will help stave off the disease if it is in his future. And he has come to terms with his genetics.

“You play the cards you are dealt,” he said.

The Days Get Harder

In San Diego, Marjie Popkin said her memory problems had gotten steadily worse in the year since she first saw Dr. Rafii.

For example, she says, she has two cats. “I have to remember when I walk out that door that they can’t come with me.”

She used to read “all the time.” Now, she says, reading is difficult. She depends on a friend, Taffy Jones, who took her to her appointment with Dr. Rafii, and who visits often and calls her every day.

But that is hard for Ms. Jones.

In many respects, Ms. Jones said, Ms. Popkin is perfectly normal. She remembers to feed her cats, she changes their litter box every day, she showers.

“Other things she is not able to deal with at all,” Ms. Jones said. Getting dressed has become a problem, and Ms. Jones has to call Ms. Popkin every morning and every night to remind her to take her pills. Ms. Popkin can no longer drive and relies on Ms. Jones to help with routine things, like getting groceries. Helping Ms. Popkin has become a time-consuming chore.

Ms. Popkin is all too aware of the situation she is in, dependent on the kindness of neighbors and Ms Jones.

“I am trying to adjust, but it’s not easy,” Ms. Popkin said in a telephone conversation. “I am pretty pragmatic. I know what the score is.”

Sometimes she sits in her apartment and just cries and cries. She has no family, and Ms. Jones is her only remaining friend; the others have drifted away.

The diagnosis of early-stage Alzheimer’s disease was a shock, Ms. Popkin said, like “a punch in the stomach.”

“This brain’s been with me since I was born — how can it change like that? Sometimes I have to think, ‘Is this really happening to me?’ ”

Her only consolation, she says, is that her father, her last remaining family member other than a cousin in North Carolina, died a few years ago, before she got the diagnosis. “He would have been devastated.”

And Ms. Popkin — is she glad now that she found out what is wrong?

“I wish I didn’t know,” she said.

    Early Tests for Alzheimer’s Pose Diagnosis Dilemma, NYT, 17.12.2010, http://www.nytimes.com/2010/12/18/health/18moral.html

The Latest Health Care Decision

December 13, 2010
The New York Times

It was no great surprise that a federal district judge in Virginia, nominated by President George W. Bush, declared a provision of the health care reform law unconstitutional. Yet his decision offers at least some hope for health care reform because it bends over backward to limit the scope of his ruling in two important respects.

The core of his ruling is that a requirement in the law that people buy health insurance in 2014 or pay a penalty is unconstitutional because it exceeds Congressional powers to regulate interstate commerce or to impose taxes for the general welfare. Two other district court judges nominated by President Bill Clinton — in Michigan and in another part of Virginia — have ruled the mandate constitutional.

Judge Henry Hudson concluded that Congress can’t regulate “economic inactivity,” the failure to buy health insurance, as if it were “economic activity” that affected interstate commerce. Yet it seems clear that decisions not to buy insurance will, in the aggregate, affect costs in the broader health care markets. We hope higher courts will find that a decision to forgo insurance simply shifts much of the cost for subsequent illness to hospitals, doctors and insured individuals. Taxpayers’ costs would rise to pay for billions of dollars in uncompensated care given to individuals who can’t pay for it.

Judge Hudson also ruled that the penalties for failing to buy insurance, though administered through the Internal Revenue Service, were really a penalty not a tax and thus can’t be justified by Congress’s authority to raise taxes for the general welfare. Yet there are precedents that seem to suggest that penalties can be considered taxes if they raise some revenue.

Virginia’s attorney general had asked the judge to invalidate the entire law if he found the mandate to buy insurance unconstitutional, but Judge Hudson invalidated only the mandate. He said he was following a time-honored rule to “sever with circumspection” by removing only problematic parts of a law.

The attorney general had also asked the judge to stop implementation of the law until a higher court rules on its constitutionality. Judge Hudson sensibly denied that request in part because the crucial provisions of the mandate, the only issue he was addressing, don’t take effect until 2013. Preparatory steps are not irreversible and states should not hang back while this case is being appealed and likely decided by the Supreme Court.

    The Latest Health Care Decision, NYT, 13.12.2010, http://www.nytimes.com/2010/12/14/opinion/14tue2.html

Health Care and the Deficit

December 11, 2010
The New York Times

Here is a basic truth about the deficit: In the long run, it cannot be fixed, without reining in spending on Medicare and Medicaid.

This year, Medicare, Medicaid and a related children’s health insurance program will account for more than 20 percent of all federal spending — higher than Social Security or defense. Unless there are big changes, by 2035 federal health care spending — driven by rising medical costs and an aging population — is projected to account for almost 40 percent of the budget.

Two bipartisan commissions have issued recommendations to sharply reduce annual deficits, in part through bold changes — some sound, others dubious — in the way health care is paid for. Here are some of the issues that Congress will need to evaluate:

COST-SHIFTING The most disturbing element of both reports is that, in their efforts to show quick savings, they shift much of the burden from the federal budget to individuals or, in some cases, to states. That may make the federal deficit look better, but it is a shell game that produces no real reduction in the cost of health care.

Both commissions would save the government significant money by forcing all but the poorest Medicare beneficiaries to pay higher premiums and co-insurance.

The White House commission, headed by Erskine Bowles and Alan Simpson, proposes to wring nearly $400 billion from health care spending between 2012 and 2020, of which the biggest single element — $110 billion — would come from increased cost-sharing by Medicare beneficiaries. The second commission, an independent panel headed by Pete Domenici and Alice Rivlin, seeks to save $137 billion from Medicare cost-sharing.

Forcing beneficiaries to pay more is a reasonable way to get them to think twice before undergoing an expensive test that may not be critically necessary. But there is a big risk that people on modest incomes might forgo needed care. Beneficiaries might have to pay hundreds or even thousands of dollars more out of pocket.

It would make sense to require wealthier older people to pick up more of the cost of their own health care. But almost half of all Medicare beneficiaries live on low incomes — below $21,000 a year for an individual and $28,000 for a couple. Cost-shifting must not be allowed to undermine the health or financial security of elderly or disabled Americans of modest means.

END OF A TAX BREAK The Domenici-Rivlin panel, the more aggressive on health care, would also phase out the exclusion that exempts workers from paying taxes for employer-subsidized insurance, a benefit that also encourages excessive use of medical care. The long-term gain in tax revenue could be huge — more than $3 trillion between 2012 and 2030 and almost $10 trillion by 2040.

The health care reform law already limits the tax exclusion for the most costly employer plans, and eliminating it entirely sounds like a good idea in principle. But it would increase the financial burden on middle- and lower-income employees. That is why they would need to have access to competitive insurance exchanges that will open in 2014 and, depending on their need, federal subsidies. Such subsidies could amount to roughly half of the tax revenues gained by eliminating the exclusion, but the net reduction of the deficit would still be large.

VOUCHERS FOR MEDICARE The Domenici-Rivlin panel has a far-reaching proposal to give Medicare enrollees vouchers to buy coverage from Medicare or a competing private plan offered on a Medicare exchange. The voucher would increase in value at roughly half the likely rate of medical inflation. If the cost of coverage rose faster than that, the beneficiary would have to pay an extra premium to cover the difference or seek a cheaper plan.

The commission believes that competition on the exchanges will cause insurance plans to find ways to lower premiums. It also believes beneficiaries will restrain their own spending. The panel projects savings from premium support and its near-term cuts and cost-shifting could be huge — more than $2 trillion through 2030 and more than $7 trillion through 2040.

We see considerable merit in having traditional Medicare and private plans compete for business, but only if the competition is fair. There would have to be tight regulation to require plans to accept all comers, to set limits to cost-sharing.

SPENDING CAPS The health care reform law already seeks to cap the growth in Medicare spending per beneficiary to roughly half the rate it has been increasing in recent decades. It empowers a new board to find savings should the target be breached, subject to Congressional veto. The Bowles-Simpson commission would expand that approach by placing a cap on total federal spending for health care — not just Medicare and Medicaid but the subsidies on new exchanges and tax exemptions. But the commission punts on what to do should the growth cap be exceeded, as many experts deem likely.

FIXING THE SYSTEM The best way to lower health care spending is to reform the dysfunctional health care system whose costs seem unrelated to the quality of care delivered. The reform law makes a good start, sponsoring research to determine which treatments are effective and which are not, starting pilot projects to change the way care is delivered and paid for, and setting up new organizations to rush successful approaches into wide use in Medicare and ultimately the private sector.

Neither commission goes much further, probably because reformers had already scooped up most of the plausible approaches. The White House commission wants to speed up the adoption rate for reforms shown to work in pilot projects, an idea we heartily endorse.

Lawmakers from both parties need to take a serious look at the commission’s recommendations. So far, we have not heard much more than the usual posturing from either side of the aisle. Meanwhile the Republicans are still vowing to repeal the new health care reform law or block its implementation. That is yet one more reason why their claims to fiscal rectitude are so unbelievable.

    Health Care and the Deficit, NYT, 11.12.2010, http://www.nytimes.com/2010/12/12/opinion/12sun1.html

Making Disability Work

December 9, 2010
The New York Times
By PETER ORSZAG

I will begin a new job for Citigroup in January, so this is my last article as a contributing columnist for The Times. I hope to see you again from time to time on the Op-Ed page.

•

One of the gravest dangers posed by the weak economy is that the unemployed will become discouraged and give up looking for work, perhaps permanently as their skills atrophy. This would be harmful not only to the workers and their families, but also to the economy as a whole, as those people would no longer contribute to economic growth. The longer the labor market remains sluggish, the more pronounced this risk becomes.

Unfortunately, at this point more than six million people have been unemployed for six months or longer. More than one million have already given up looking for work because they believe no job is available. And a drastic rise in applications for disability insurance suggests we may be headed for more long-lasting trouble. The number of disability applications has reached more than 750,000 a quarter, according to the Social Security Administration, an increase of more than 50 percent from four years ago.

The disability insurance program provides crucial support for people who can no longer work because of a disability. But once someone begins receiving benefits, the likelihood that he will re-enter the work force is almost nonexistent; recipients become permanently dependent on the program.

The result is not only lost economic productivity, but also a fiscal burden for the federal government: disability benefits now cost more than $120 billion a year, and Medicare benefits for those on disability add $70 billion.

The spike in disability insurance applications (and awards) does not reflect a less healthy population. The fraction of working-age adults who report a disability, about one in 10, has remained roughly constant for the past 20 years. (Indeed, it would be surprising if the number of workers with disabilities had risen by 50 percent over the past four years.) Rather, the weak labor market has driven more people to apply for disability benefits that they qualify for but wouldn’t need if they could find work.

When Congress created the disability insurance program in 1956, it required that recipients be unable to “engage in substantial gainful activity in the U.S. economy.” In other words, they had to be unable to work. That was sensible at the time, when more jobs involved physical labor and technologies to assist people with disabilities were not widely available.

Today, however, many people with disabilities are able to engage in some form of work — even if they can’t admit that and still keep their insurance benefits. Cutting off access to the workplace in this way is both unfortunate and unnecessary — and reinforces the threat that the current downturn could cause a long-term reduction in the share of people who work.

So what should be done?

First, macroeconomic policy. We need more stimulus immediately, and more deficit reduction enacted now to take effect in two or three years. The plan just proposed by the White House in a compromise with Congressional Republicans is encouraging in that it includes a new payroll tax holiday, a helpful stimulus. It does not reduce future deficits, but at least it avoids making the Bush tax cuts permanent, reserving the flexibility to address medium-term deficits down the road.

Even if this plan goes ahead, however, the unemployment rate is likely to remain high for some time. For it to fall by even one percentage point (from 9.5 percent to 8.5 percent) the economy needs to grow by about 4.5 percent a year.

Second, unemployment insurance should be extended, as President Obama’s compromise plan also would do. Unemployment benefits are a form of stimulus: they spur spending and thereby help keep the economy afloat. Just as important, unemployment benefits keep many people from falling back on disability insurance — and unlike disability insurance, which effectively prohibits beneficiaries from seeking work, unemployment insurance requires recipients to keep looking for a job and thus remain connected to the work force.

Finally, the disability insurance program itself must be reformed. Program administrators understand the need to encourage beneficiaries to return to work, and they have experimented with various incentives. Such initiatives have generally been ineffective, though, because they reach beneficiaries too late, after they have already become dependent on the program and lost their attachment to the work force.

A better approach has been suggested by David Autor of M.I.T. and Mark Duggan of the University of Maryland. In a paper released last week from the Center for American Progress and the Hamilton Project, these economists argue that employers should be required to offer their workers private disability insurance. Such coverage would provide people who have a work-limiting disability with vocational assistance, workplace accommodation and limited wage replacement. All of these benefits would kick in within 90 days of the onset of disability, to avoid the problems with delayed assistance that have plagued efforts to reform public disability insurance. Private employers would have an incentive to prevent their workers from having to file disability applications, because their insurance premiums would rise in response to higher disability rates.

Disabled workers could remain on this privately financed insurance for two years, and then be eligible for the existing public program. The goal would be to minimize long-term dependency, and re-orient the federal disability insurance program toward assisting those who are truly unable to work.

One concern is that this approach would burden firms with additional human resource costs when we need to encourage hiring. But the costs are projected to be modest — roughly $250 per worker per year. And if they help to reduce the future payroll tax increases that would be needed to finance rapid growth in disability benefits, the pressure on overall labor costs would be even smaller.

Another concern is that private insurance firms would need to be given substantially expanded responsibility for evaluating workers’ disabilities. Mr. Autor and Mr. Duggan propose to mitigate this potential problem by suggesting that workers be allowed to appeal any such evaluations to state government agencies.

The Netherlands has adopted a program like this, and the results so far are promising. In 1994, the Dutch government required all firms to finance the first six weeks of disability benefits. That period was later extended to one year and then to two years. In 2002, the program was broadened to require back-to-work plans, developed cooperatively by the disabled worker, his employer and a consulting doctor. The number of disability recipients in the Netherlands has since declined significantly.

None of these policy changes would be easy. But failing to act would result in millions of Americans needlessly dropping out of the work force. In our precarious economy, neither progressives nor conservatives should be willing to watch passively as the disability insurance rolls grow, and beneficiaries are locked out of the labor market.

Peter Orszag, the director of the White House Office of Management and Budget from 2009 to July 2010, is a distinguished visiting fellow at the Council on Foreign Relations.

    Making Disability Work, NYT, 9.12.2010, http://www.nytimes.com/2010/12/10/opinion/10orszag.html

Arizona Cuts Financing for Transplant Patients

December 2, 2010
The New York Times
By MARC LACEY

PHOENIX — Even physicians with decades of experience telling patients that their lives are nearing an end are having difficulty discussing a potentially fatal condition that has arisen in Arizona: Death by budget cut.

Effective at the beginning of October, Arizona stopped financing certain transplant operations under the state’s version of Medicaid. Many doctors say the decision amounts to a death sentence for some low-income patients, who have little chance of survival without transplants and lack the hundreds of thousands of dollars needed to pay for them.

“The most difficult discussions are those that involve patients who had been on the donor list for a year or more and now we have to tell them they’re not on the list anymore,” said Dr. Rainer Gruessner, a transplant specialist at the University of Arizona College of Medicine. “The frustration is tremendous. It’s more than frustration.”

Organ transplants are already the subject of a web of regulations, which do not guarantee that everyone in need of a life-saving organ will receive one. But Arizona’s transplant specialists are alarmed that patients who were in line to receive transplants one day were, after the state’s budget cuts to its Medicaid program, ruled ineligible the next — unless they raised the money themselves.

Francisco Felix, 32, a father of four who has hepatitis C and is in need of a liver, received news a few weeks ago that a family friend was dying and wanted to donate her liver to him. But the budget cuts meant he no longer qualified for a state-financed transplant.

He was prepared anyway at Banner Good Samaritan Medical Center as his relatives scrambled to raise the needed $200,000. When the money did not come through, the liver went to someone else on the transplant list.

“I know times are tight and cuts are needed, but you can’t cut human lives,” said Mr. Felix’s wife, Flor. “You just can’t do that.”

Such high drama is unfolding regularly here as more and more of the roughly 100 people affected by the cuts are becoming known: the father of six who died before receiving a bone marrow transplant, the plumber in need of a new heart and the high school basketball coach who struggles to breathe during games at high altitudes as she awaits a lung transplant.

“I appreciate the need for budget restraints,” said Dr. Andrew M. Yeager, a University of Arizona professor who is director of the Blood and Marrow Transplantation Program at the Arizona Cancer Center. “But when one looks at a potentially lifesaving treatment, admittedly expensive, and we have data to support efficacy, cuts like this are shortsighted and sad.”

State Medicaid officials said they recommended discontinuing some transplants only after assessing the success rates for previous patients. Among the discontinued procedures are lung transplants, liver transplants for hepatitis C patients and some bone marrow and pancreas transplants, which altogether would save the state about $4.5 million a year.

“As an agency, we understand there have been difficult cuts and there will have to be more difficult cuts looking forward,” said Jennifer Carusetta, chief legislative liaison at the state Medicaid agency.

The issue has led to a fierce political battle, with Democrats condemning the reductions as “Brewercare,” after Gov. Jan Brewer.

“We made it very clear at the time of the vote that this was a death sentence,” said State Senator Leah Landrum Taylor, a Democrat. “This is not a luxury item. We’re not talking about cosmetic surgery.”

The Republican governor has in turn blamed “Obamacare,” meaning the federal health care overhaul, for the transplant cuts even though the Arizona vote came in March, before President Obama signed that bill into law.

But a top Republican, State Representative John Kavanagh, has already pledged to reconsider at least some of the state’s cuts for transplants when the Legislature reconvenes in January. Mr. Kavanagh, chairman of the Appropriations Committee, said he does not believe lawmakers had the full picture of the effect of the cuts on patients when they voted.

“It’s difficult to be linked to a situation where people’s lives are jeopardized and turned upside down,” he said in an interview. “Thankfully no one has died as a result of this, and I believe we have time to rectify this.”

Across the country, states have restricted benefits to their Medicaid programs, according to a 50-state survey published in September by the Kaiser Commission on Medicaid and the Uninsured. But none have gone as far as Arizona in eliminating some transplants, which are considered optional services under federal law.

Before the Legislature acted, Arizona’s Medicaid agency had provided an analysis to lawmakers of the transplants that were cut, which many health experts now say was seriously flawed. For instance, the state said that 13 of 14 patients under the state’s health system who received bone marrow transplants from nonrelatives over a two-year period died within six months.

But outside specialists said the success rates were considerably higher, particularly for leukemia patients in their first remission.

“Something needs to be done,” said Dr. Emmanuel Katsanis, a bone marrow transplant expert at the University of Arizona. “There’s no doubt that people aren’t going to make it because of this decision. What do you tell someone? You need a transplant but you have to raise the money?”

Just before the Oct. 1 deadline, Mark Price, a father of six who was fighting leukemia, learned he needed a bone marrow transplant. But his doctor, Jeffrey R. Schriber, found donor matches for his transplant the very day the new rules went into effect, and Mr. Price no longer qualified for coverage by the Arizona Health Care Cost Containment System, the formal name for the state’s Medicaid program.

What happened next was at once inspirational and heart-rending.

Out of the blue, an anonymous financial donor quickly stepped forward and agreed to cover the hundreds of thousands of dollars needed for Mr. Price’s surgery. But Mr. Price died last weekend, after his cancer returned before the operation could be done. He was buried on Thursday, next to his grandfather.

“It’s not correct to say that he died as a result of the cuts,” said Dr. Schriber, who is active in lobbying for the financing to be restored. “Did it prey on his mind? Did it make his last days more difficult? No doubt.”

Elsewhere, the fund-raising is already under way.

Mr. Felix and others are now trying to raise enough for new organs through NTAF, a nonprofit organization based in Pennsylvania formerly known as the National Transplant Assistance Fund that helps transplant patients pay for their medical costs. National coverage of their plight has already led to more than $100,000 in donations for some of the patients affected by the budget cuts. The Felix family is also planning a yard sale this weekend so he does not lose the chance to get another liver.

There has been a flurry of lobbying to persuade the state to reverse the decision. Dr. Gruessner said he and others met with state health officials recently to propose other cuts associated with transplants, like eliminating tests typically conducted before surgery.

If the Legislature does decide to reconsider the cuts, one of the affected people, a plumber and father of three named Randy Shepherd, 36, who has an ailing heart and needs a transplant, plans to attend the debate.

“I’m trying not to take it personally,” he said of being cut out of the program. “None of the politicians had heard of me when they made their decision. They didn’t say, ‘Let’s kill this guy.’ ”

    Arizona Cuts Financing for Transplant Patients, NYT, 2.12.2010, http://www.nytimes.com/2010/12/03/us/03transplant.html

Daily Pill Lowers H.I.V. Infection Risk, Study Finds

November 23, 2010
The New york Times
By DONALD G. McNEIL Jr.

In a development that could change the battle against AIDS, researchers have found that taking a daily antiretroviral pill greatly lowers the chances of getting infected with the virus.

In the study, published Tuesday by the New England Journal of Medicine, researchers found that the hundreds of gay men randomly assigned to take the drugs were 44 percent less likely to get infected than the equal number assigned to take a placebo.

But when only the men whose blood tests showed they had taken their pill faithfully every day were considered, the pill was more than 90 percent effective, said Dr. Anthony S. Fauci, head of the division of the National Institutes of Health, which paid for the study along with the Bill and Melinda Gates Foundation.

“That’s huge,” Dr. Fauci said. “That says it all for me.”

The large study, nicknamed iPrEx, included nearly 2,500 men in six countries and was coordinated by the Gladstone Institutes of the University of California, San Francisco.

The results are the best news in the AIDS field in years, even better than this summer’s revelation that a vaginal microbicide protected 39 percent of all the women testing it and 54 percent of those who used it faithfully.

Also, the antiretroviral pill — Truvada, a combination of two drugs, tenofovir and emtricitabine — is available by prescription in many countries right now, while the microbicide gel is made only in small amounts for clinical trials.

The protection, known as “pre-exposure prophylaxis” or “PreP,” is also the first new form available to men, especially men who cannot use condoms because they sell sex, are in danger of prison rape, are under pressure from partners or lose their inhibitions when drunk or high.

It is a form of protection “that does not involve getting permission from the other partner, and that’s important,” said Phill Wilson, president of the Black AIDS Institute, which focuses on the epidemic among blacks.

Michel Sidibé, the head of UNAIDS, the United Nations AIDS-fighting agency, called it “a breakthrough that will accelerate the prevention revolution.”

Because Truvada is available now, some clinicians already prescribe it for prophylaxis, Dr. Fauci said, but whether doing so becomes official policy will depend on discussions by the Centers for Disease Control and Prevention, the Food and Drug Administration, medical societies and others, which could take months.

Although the C.D.C. would prefer that doctors wait for further studies, more probably will prescribe it now that this study is out, said Dr. Kevin Fenton, chief of the agency’s AIDS division, so the C.D.C. will soon release suggested guidelines.

The agency will encourage that the drug be prescribed only with close medical supervision and used only with other safe-sex practices, treatment for venereal diseases and counseling.

“The results are encouraging, but it’s not time for gay men to throw away their condoms,” Dr. Fenton said.

AIDS advocacy groups were very excited by the results.

“If you comply with it, this works really well,” said Chris Collins, policy director of amfAR, the Foundation for AIDS Research. “This is too big to walk away from.”

Mitchell Warren, executive director of AVAC, which lobbies for AIDS prevention, called the study “a great day for the fight against AIDS” and said gay men and others at risk needed to be consulted on the next steps.

AIDS experts and the researchers issued several caveats about the study’s limitations.

It was only of gay men and only of one drug combination. More studies, now under way, are needed to see if they duplicate these results and to see whether Truvada also protects heterosexual men and women, prostitutes and drug users who share needles, and whether other drugs will work, too.

There is no medical reason to think the pill would not work in everyone, since it attacks the virus in the blood, rather than in the vaginal wall as a microbicide does.

Different regimens, like taking the pills only when sex is anticipated instead of daily, also need testing.

Also, many men in the study failed to take all their pills, and some clearly lied about it. For example, some who claimed to take them 90 percent of the time had little or no drug in their bloodstreams.

Although the pills caused no major side effects in the study, some men disliked the relatively minor ones, like nausea and headaches. Also, as is common in clinical trials, some stopped bothering once they suspected they might be taking a placebo.

“People have their own reasons,” Mr. Collins said. “People don’t take their Lipitor every day either.”

A major question now is who will pay for the drug.

In the United States, Truvada, made by Gilead Sciences, costs $12,000 to $14,000 a year. In very poor countries, generic versions costs as little as 40 cents a pill.

Globally, only about 5 million of the 33 million people with AIDS are on antiretrovirals, and, in an era of tight foreign-aid budgets, that number is not expected to rise quickly.

Hundreds of millions of Africans, eastern Europeans and Asians are at risk and could benefit from prophylaxis, but that would cost tens of billions of dollars.

If he had the money, Mr. Sidibé of UNAIDS said, he would target high-risk groups like sex workers, gay men, drug users and uninfected people married to infected people.

In this country, insurers and Medicare normally pay for the drugs, and the Ryan White Act covers the cost for the poor — but none of these payers yet have policies on supplying the drugs to healthy people.

One fear some scientists have is that putting more people on the drugs will speed the evolution of drug-resistant strains.

None of the 2,499 participants developed resistance to tenofovir. Three were found to have strains resistant to emtricitabine, but investigators believe all three were infected before the study began, but at levels low enough to have been missed by their first H.I.V. tests.

Because participants were tested monthly and those who got infected were put on triple therapy cocktails, it was unlikely any were on two-drug Truvada long enough to develop drug resistant strains.

Another fear was that the participants would become so fearless that they would stop using condoms, but the opposite effect was seen — they used condoms more often and had fewer sex partners. But that can also be a function of simply being enrolled in a study and getting a steady diet of safe sex advice and free condoms, the investigators said.

The study took place at 11 sites in the United States, South Africa, Brazil, Thailand, Ecuador and Peru. Other trials of pre-exposure prophylaxis have about 20,000 volunteers enrolled around the world. Their results are expected to arrive in a steady stream over the next two years.

    Daily Pill Lowers H.I.V. Infection Risk, Study Finds, 23.11.2010, http://www.nytimes.com/2010/11/24/health/research/24aids.html

G.O.P. Plans to Use Purse Strings to Fight Health Law

November 6, 2010
The New York Times
By ROBERT PEAR

WASHINGTON — As they seek to make good on their campaign promise to roll back President Obama’s health care overhaul, the incoming Republican leaders in the House say they intend to use their new muscle to cut off money for the law, setting up a series of partisan clashes and testing Democratic commitment to the legislation.

Republicans, who will control the House starting in January but will remain in the minority in the Senate, acknowledge that they do not have the votes for their ultimate goal of repealing the health law, the most polarizing of Mr. Obama’s signature initiatives.

But they said they hoped to use the power of the purse to challenge main elements of the law, forcing Democrats — especially those in the Senate who will be up for re-election in 2012 — into a series of votes to defend it.

Republican lawmakers said, for example, that they would propose limiting the money and personnel available to the Internal Revenue Service, so the agency could not aggressively enforce provisions that require people to obtain health insurance and employers to help pay for it. Under the law, individuals and employers who flout the requirements will face tax penalties.

Moreover, Republican leaders said, they plan to use spending bills to block federal insurance regulations to which they object. And they will try to limit access to government-subsidized private health plans that include coverage of abortion — one of the most contentious issues in Congressional debate over the legislation.

Those are just a few examples of the ways in which newly empowered House Republicans plan to use spending bills to pressure Mr. Obama and Senate Democrats to accept changes in the law.

Given their slim majority, Senate Democrats must stick together if they want to avoid sending Mr. Obama spending bills and other legislation that he would feel compelled to veto, setting up the prospect of a broader deadlock and, in an extreme situation, a government shutdown.

The House Republican whip, Representative Eric Cantor of Virginia, described the strategy this way: “If all of Obamacare cannot be immediately repealed, then it is my intention to begin repealing it piece by piece, blocking funding for its implementation and blocking the issuance of the regulations necessary to implement it.”

“In short,” Mr. Cantor said, “it is my intention to use every tool at our disposal to achieve full repeal of Obamacare.”

The Senate Republican leader, Mitch McConnell of Kentucky, said he, too, wanted to shut off money for the new law.

Mr. Obama has made clear that he will fight to preserve all the fundamental elements of the law. When asked if the president would veto legislation to cut off money, his spokesman, Robert Gibbs said, “I don’t think we’ll get to that.”

Both sides said they were determined to avoid a government shutdown like the one in 1995 that, by many accounts, did political damage to House Republicans and Newt Gingrich, who was then speaker.

Anticipating the Republican assault, White House officials said Mr. Obama would emphasize how the law protects consumers and gives them more control of their insurance. Administration officials are working with Senate Democrats to arrange hearings at which consumers would explain how they have already benefited from the law.

One of the president’s strongest allies is Senator Tom Harkin, Democrat of Iowa, an architect of the law. Mr. Harkin said he would “fight any attempt to defund the law or repeal its consumer protections.” He is well placed to lead such resistance. He is chairman of the Senate’s health committee and of its Appropriations subcommittee responsible for health programs.

The number and variety of restrictions Congress can impose in spending bills is almost unlimited. A bill passed by the House last year, for example, stipulated that no federal money could be used to buy light bulbs unless they met certain energy efficiency standards. The same bill said, “No funds appropriated in this act may be used for the transportation of students or teachers in order to overcome racial imbalance in any school.”

House Republicans could easily pass similar provisos stating that no federal money could be used to carry out specific sections of the new health care law.

By attaching the restrictions to appropriations bills, House Republicans can force negotiations with the Senate. The Hyde amendment, restricting the use of federal money to pay for abortion, began as such a rider more than 30 years ago.

House Republicans said their efforts were inspired, in part, by the words of Senator Robert C. Byrd, the West Virginia Democrat who died this year. Mr. Byrd described the power of the purse as “one of the most effective bulwarks ever constructed” to shackle the hands of an overreaching executive.

Even if judges uphold the constitutionality of the law, federal officials will still need money to administer and enforce it. And that is where House Republicans see an opportunity to assert their influence, with a real possibility of a stalemate.

The White House has provided money to states to help them get ready — to scrutinize increases in insurance premiums and to set up regulated markets known as insurance exchanges. In addition, the law provided $1 billion for “federal administrative expenses.” But that is far less than will be required.

The Congressional Budget Office says the Internal Revenue Service will need $5 billion to $10 billion over 10 years to determine who is eligible for tax credits and other subsidies intended to make insurance affordable. The Department of Health and Human Services will need at least that much to carry out changes in Medicaid, Medicare and the private insurance market, the budget office said.

The law provided $11 billion for community health centers to serve 20 million more low-income people, including many expected to gain coverage under the law. Many Republicans, including President George W. Bush, have supported such clinics.

But Daniel R. Hawkins Jr., senior vice president of the National Association of Community Health Centers, said, “It’s unclear how we will fare in the new climate.”

The House Republicans’ campaign manifesto proposed “strict budget caps” that would cut spending for most domestic programs subject to annual appropriations.

The conflict over health care may be the biggest obstacle to cooperation between Mr. Obama and Republicans in Congress.

“House Republicans cannot enact legislation the president won’t sign,” said R. Scott Lilly, a former Democratic staff director of the House Appropriations Committee. “But the president cannot force them to appropriate money they don’t want to appropriate.”

    G.O.P. Plans to Use Purse Strings to Fight Health Law, NYT, 6.11.2010, http://www.nytimes.com/2010/11/07/health/policy/07health.html

Stepping In for a Parent With Alzheimer’s

November 5, 2010
The New York Times
By TARA SIEGEL BERNARD

As my grandfather’s mind slipped away, first slowly, then quickly, as Alzheimer’s disease moved in, he tried to be reassuring. “I remember the important things,” he would tell us.

And he did. He may not have remembered that Barack Obama was president or that he just had a bowl of ice cream (my kindergartner nephew took full advantage), but he always remembered his family.

Money management was another matter. Early on in the disease, small red flags began to appear — failure to pay the association fees on the condominium in Florida, overpaid bills or repetitive trips to the A.T.M. Over time, he had to hand off most financial responsibilities to my mother and grandmother.

My grandfather was lucky enough to have relatives nearby who were able to gently intervene before the small mistakes escalated into something more serious, which is all too common.

But family members need to carefully consider how they approach their new role as financial caretaker. For adult children, this is the beginning of the role reversal. You may be stepping up to handle mundane tasks like paying the bills, but it’s also the time when you begin to think about your parent’s mortality, and perhaps your own.

For the person with encroaching dementia, the loss of autonomy can be devastating. “What often occurs is that the elder loses additional self-esteem, becomes more depressed and in turn becomes less active,” said Daniel C. Marson, a neuropsychologist at the University of Alabama at Birmingham.

And that’s why these matters must be handled delicately — and before a more serious financial unraveling occurs — whether the individual simply needs you to check in once a month, or you need to become the chief financial officer. Below are some strategies.

 

THE TALK Discussing money matters with someone who is already dealing with diminished mental capacity can be challenging, though the conversation can unfold in many ways. It all depends on the family dynamics and the elder person’s level of impairment, personality and comfort level with financial issues, said Dr. Marson, who is also director of the university’s Alzheimer’s Disease Center.

In certain situations, the best place to start a dialogue may be in the doctor’s office. “Go to a health care professional who can be supportive and understand some of the dynamics going on, so that the conversation is structured around identifying something that is a need and that we don’t want to get any worse,” said Becky Bigio, director of the Senior Source program at Selfhelp Community Services in New York.

The needs of the person with dementia will change over time, which means the financial caretaker’s role will also need to evolve. It may help to follow a slow but natural progression. “Involvement may go from, ‘Do you have enough money to pay the bills?’ to ‘It’s O.K. to pay that bill,’ to ‘I’ll just pay the bills now,’” said Tom Davison, a financial planner in Columbus, Ohio, who has worked with several elderly people in his family and practice. “The changes may be fast or slow, but move at their pace.”

 

SAFETY MECHANISMS There are also several ways to keep a watchful yet unobtrusive eye on a parent’s financial affairs, even if you don’t live nearby. Setting up automatic bill payments may be the most obvious way. For parents who want to continue paying the bills, allow them to write checks for services that won’t cause harm if the payment is late, and that you can easily remedy later.

Some major banks said they allowed customers to authorize a third party to receive statements — for mortgages, loans or credit cards — which makes it easy to spot a missed payment or unusual activity. (In some cases, the bank may require a power of attorney or notarized consent.) Long-term care insurers, like John Hancock and Genworth, also permit policyholders to have another person receive a notice if a bill is past due or if there is a lapse in coverage.

Other companies should follow that lead and make it easy to set up an automatic e-mail notification of a late notice.

 

LEGAL DOCUMENTS Proper legal documents should also be in place. A durable power of attorney enables a person to step in and handle a parent’s financial affairs. Most banks are understandably wary of powers of attorney, given the room for fraud and abuse. So check with your institution about its requirements.

Opening a bank account in the name of a trust also gives one or more trustees decision-making and check-writing privileges. The same goes for anything else in a revocable living trust, where accounts and assets are titled to the trust. “Trusts like these can have two or more trustees at the same time and can be set up to operate individually or must act together — there’s all kinds of flexibility and can be written to respond to the particular circumstances of an individual,” Mr. Davison said. “Do they want checks and balances between an attorney and a kid? Want two kids to have to agree and both sign stuff?” Assets in the living trust have the added benefit of bypassing probate after the person dies.

It may be easier to simply add an adult child’s name to the account, but consult with an adviser to make sure it won’t interfere with the person’s estate plans.

 

SIMPLIFY AND SET ROUTINES It’s less confusing for the financial caretaker and the elderly person to keep track of just a few accounts. So try to consolidate. Experts also recommend setting up routines and identifying the triggers that make the elderly person uneasy. For Mr. Davison’s elderly aunt, who had short-term memory loss, the trigger was the mail. As a former business manager who balanced her books to the penny, she was often overwhelmed when the mail arrived.

So Mr. Davison told her to open the mail every day and put it in a folder. Each weekend, he and his wife would visit and review the mail with his aunt, a routine that put her at ease. “See what the trigger points are, and try some ways to soften them,” he said. “You often can’t solve them, but you can soften them. And whatever was helpful three months ago may not be helpful now. It’s a progression.”

 

HIRING HELP You obviously need to investigate any provider when dealing with the vulnerable and easily swindled, but there are several places to begin your search. AARP offers a money management program for lower-income people in some parts of the country, or, you can hire an accountant or another party to handle the finances. The American Association of Daily Money Managers is another option, and you can call to request someone who has experience with helping people with dementia.

There are also companies that cater to elderly people’s special needs. Wells Fargo Private Bank, which requires more than $1 million in investable assets, offers an Elder Services program. It will coordinate a variety of services, from dealing with medical claims, taking the cat to the veterinarian and paying the bills, while keeping a close eye on the accounts for fraudulent activities. The service is included in the bank’s investment management fees, which range from 0.80 to 2 percent of assets. Let’s hope we see more such services — and not just for the wealthy.

 

PLAN NOW Ideally, all families should prepare for potential issues while everyone is still healthy. There are generally three questions families should answer, according to Ken Dychtwald, a gerontologist, psychologist and consultant. Who will handle the finances? What are the individual’s preferences on long-term care? And how will we pay for it?

“The biggest worry that people have about growing old is being a burden on their family,” Mr. Dychtwald said. “Ironically, if you don’t think about a game plan before it’s needed, then not only does it become a burden, but there is often damage that comes with it,” both financial and emotional.

My mother now says she believes the conversation about money matters should be a rite of passage of sorts, for everyone who reaches retirement. But it’s never too soon to have the talk.

    Stepping In for a Parent With Alzheimer’s, NYT, 5.11.2010, http://www.nytimes.com/2010/11/06/your-money/06money.html

Looking for a Superbug Killer

November 5, 2010
The New York Times
By ANDREW POLLACK

Worried about an impending public health crisis, government officials are considering offering financial incentives to the pharmaceutical industry, like tax breaks and patent extensions, to spur the development of vitally needed antibiotics.

While the proposals are still nascent, they have taken on more urgency as bacteria steadily become resistant to virtually all existing drugs at the same time that a considerable number of pharmaceutical giants have abandoned this field in search of more lucrative medicines. The number of new antibiotics in development is “distressingly low,” Dr. Margaret A. Hamburg, commissioner of the Food and Drug Administration, said at a news conference last month. The world’s weakening arsenal against “superbugs” has prompted scientists to warn that everyday infections could again become a major cause of death just as they were before the advent of penicillin around 1940.

“For these infections, we’re back to dancing around a bubbling cauldron while rubbing two chicken bones together,” said Dr. Brad Spellberg, an infectious disease specialist at Harbor-U.C.L.A. Medical Center in Torrance, Calif.

For example, scientists have become alarmed by the spread from India of a newly discovered mutation called NDM-1, which renders certain germs like E. coli invulnerable to nearly all modern antibiotics. About 100,000 Americans a year are killed by infections acquired in hospitals, many resistant to multiple antibiotics. Methicillin-resistant staphylococcus aureus, or MRSA, the best known superbug, now kills more Americans each year than AIDS.

While the notion of directly subsidizing drug companies may be politically unpopular in many quarters, proponents say it is necessary to bridge the gap between the high value that new antibiotics have for society and the low returns they provide to drug companies.

“There is a market failure,” said Representative Henry A. Waxman, a California Democrat and the chairman of the House Energy and Commerce Committee, who said he was considering introducing legislation. “We need to look at ways to spur development of this market.”

Mr. Waxman will lose his committee chairmanship with the Republicans having won control of the House this week. But the idea of spurring antibiotic development appears to have some bipartisan support. Representative Phil Gingrey, a Georgia Republican and a physician, recently introduced the Generating Antibiotic Incentives Now bill, which would provide certain antibiotics with five extra years of protection from generic competition and speed the reviews of new antibiotics by the Food and Drug Administration.

Besides tax breaks and extra protection from competition, other ideas policy makers are considering include additional federal funding of research and guaranteed purchases by the government of new antibiotics. Measures like these are already used to encourage the development of drugs for rare diseases, through the Orphan Drug Act, and for illnesses like malaria that primarily afflict poor countries.

The Obama administration is also taking some steps. The federal agency that oversees development of treatments for bioterrorism agents like anthrax is broadening its scope to encompass more common infections. In August, the agency, known as the Biomedical Advanced Research and Development Authority, awarded its first such “multi-use” contract, giving an initial $27 million to a company called Achaogen to develop an antibiotic that could be used for plague and tularemia as well as antibiotic-resistant infections.

The Department of Health and Human Services is considering creating an independent fund that would invest in small bio-defense companies. Antibiotic-resistant germs would be one priority, according to a report that the department issued in August.

The European Union is also working on a plan, based on proposals from the London School of Economics. A year ago, the United States and the European Union formed a task force on antibiotic resistance.

Despite the activity, there is no consensus on what would work best and little discussion of how much such measures would cost.

A paper issued last month by the Office of Health Economics, a consulting firm owned by the British pharmaceutical industry’s trade group, suggested that incentives exceeding $1 billion per drug would be required.

Some critics say the case for incentives is not yet persuasive. There are signs that the drug industry is picking up its efforts on its own, in response to perceived need. The number of antibiotics in clinical trials has climbed sharply in the last three years, reversing a steady decline that began in the 1980s, according to figures from the F.D.A. The efforts are being led by small companies, which can be satisfied with smaller sales.

Ramanan Laxminarayan, who directs the Extending the Cure project on antibiotic resistance at Resources for the Future, a policy organization, said the government should focus on conserving the effectiveness of existing antibiotics. That could be done by preventing unnecessary use in people and farm animals and requiring better infection control measures in hospitals.

“There’s not a recognition yet that we should think about antibiotics as a natural resource and we should conserve them like we do fish,” Mr. Laxminarayan, an economist, said. Kevin Outterson, an associate professor of law at Boston University, said one way to encourage both new development and conservation would be to pay drug companies to develop new antibiotics but not to aggressively market them. Incentives, he said, “must be conditioned on the companies’ changing their behavior.”

Only five new antibiotics were approved by the F.D.A. from 2003 through 2007, down from 16 in the period from 1983 to 1987. A survey last year by European health authorities found only 15 antibiotics in clinical trials that offered some promise of going beyond what is available today.

Only five of the 13 biggest pharmaceutical companies still try to discover new antibiotics, said Dr. David M. Shlaes, a consultant to the industry and the author of a new book “Antibiotics: The Perfect Storm.”

One reason is that antibiotics are typically taken for a week or two and usually cure the patient. While that makes them cost-effective for the health system, it also makes them less lucrative to drug companies than medicines for diseases like cancer or diabetes, which might be taken for months, or even for life, because they do not cure the patient.

“There’s this perverse disincentive against antibiotics because they work so well,” said J. Kevin Judice, chief executive of Achaogen.

Another factor is that new antibiotics are likely to be used only sparingly at first, to stave off the emergence of resistance. While that might be medically appropriate, it reduces the ability of a drug company to recoup its investment, said Dr. Barry I. Eisenstein, a senior vice president at the antibiotic maker Cubist Pharmaceuticals. Another factor discouraging investment, some experts say, is that the F.D.A. recently made it harder for new antibacterial drugs to win approval.

Leading the call for incentives has been the Infectious Diseases Society of America, whose members are infectious disease specialists. It is calling for a “10 by ‘20” initiative to develop 10 new antibiotics by 2020. The initiative, which is more an aspiration than a plan, has been endorsed by numerous other medical societies.

But so far there is little consumer support. “We don’t have any patient groups for Acinetobacter,” said Robert J. Guidos, the society’s vice president for public policy and government relations, referring to a drug-resistant bacterium. Patient groups concerned about superbugs tend to focus on reducing the spread of infections in hospitals.

The Pharmaceutical Research and Manufacturers of America, the main trade group for big drug companies, has not taken a position on incentives because not all members are in the antibiotic business, said David R. Brennan, the chief executive of AstraZeneca and former chairman of the trade group.

Mr. Brennan, whose company is still in the antibiotic business, said that at a minimum, new antibiotics should be given longer protection from generic competition to make up for the fact that they are used sparingly when they go on sale. “Give us more time at the back end,” he said.

    Looking for a Superbug Killer, NYT, 5.11.2010, http://www.nytimes.com/2010/11/06/health/policy/06germ.html

As H.I.V. Babies Come of Age, Problems Linger

November 5, 2010
The New York Times
By PAM BELLUCK

WARWICK, R.I. — “They’ve been telling me since age 3 that I would die,” Tom Cosgrove said quietly. “Then age 6, age 8, age 10.”

Now 20, he is considered the longest-living person born with H.I.V. in his state, but every year has brought struggle.

As a toddler at a shelter for children infected with H.I.V. from birth, he watched others die. Then, AIDS killed his mother and newborn brother. At 8, his body rejected medication and he became temporarily unable to walk.

He raged with anger, once even striking a teacher with a chair. Classmates, paranoid about his disease, refused to shake his hand or sit at his lunch table. Friends’ parents forbade them to visit, and he could not join basketball teams or karate classes.

Even now, medications impair his short-term memory, making school, and job prospects, difficult.

“We call them his stupid drugs,” said Barbara Cosgrove, who adopted Tom at 3. “But, as I say to Tom, ‘You’re either stupid or you’re dead.’ ”

At a time when H.I.V. in the United States has become a manageable disease for many, Tom Cosgrove and others like him are proof of the epidemic’s troubling, lingering legacy. They are the survivors, born beginning in the 1990s to the first big wave of people with AIDS, babies practically destined to die. Improvements in drugs, along with some luck, allowed some 10,000 of them to live — and these days only about 200 children a year are born with H.I.V., thanks to vigilant drug treatment of infected pregnant women.

But life for those first H.I.V. babies now entering adolescence and adulthood has been a battle, and their experience is considered so significant — not only in this country but also for the millions of H.I.V.-positive babies worldwide — that federal health agencies have begun an extensive study to follow these young people as they grow up.

Some are weakened by years of yo-yoing symptoms that early drugs failed to treat. Some have developmental delays or other problems related to having H.I.V. at birth. And their medications often have harsher side effects than those taken by people infected more recently as teenagers or adults because complications from their illness, or previous drugs they took and became resistant to, have made their disease more stubborn to treat.

Emotionally, they grapple with hostility toward parents who infected them, grief that those parents suffered and usually died, and anxiety about trusting others with a secret that still provokes hazing and fear.

And a serious problem is emerging: some are rebelling or asserting independence by skipping or stopping medication, which can make H.I.V. spiral out of control and become impervious to previously effective therapies.

“It ain’t over yet,” Dr. Ellen Cooper, medical director of pediatric and adolescent H.I.V. at Boston Medical Center, said about keeping these young people alive and healthy. Although she has not lost a patient in five years, she said, “I’m expecting a second wave” of these young people “dying because they’re not adherent” to medication, or because of “complications from treatment.”

Dr. Lynne M. Mofenson, chief of pediatric, adolescent and maternal AIDS at the National Institute of Child Health and Human Development, said that despite H.I.V. babies’ increased survival, their “mortality is still thirtyfold higher than similarly aged children,” and there is “a lot of research that’s needed, and interventions to improve their lives.”

Seven agencies of the National Institutes of Health are following 451 H.I.V. babies ages 7 to 16, monitoring their hearts, cholesterol, bones, brains, hearing, sexual development, school performance, language ability, behavior and mental health.

Preliminary findings show many of their current lifesaving drugs cause high cholesterol, raising fears of serious heart problems, said Dr. Russell Van Dyke, the study’s co-principal investigator. Their bone density appears poor. And many have mental health and behavioral problems, although it is unclear to what degree those problems are related to the disease or to the children’s often-difficult family circumstances.

“There is a lot of concern,” Dr. Van Dyke said, “about how the kids adapt to living and what sort of challenges they have. The lessons are going to be applicable to the rest of the world.”

‘I Was Born This Way’

Davi Morales is the kind of young person doctors worry about. He has H.I.V.-related cognitive disabilities, and spent months homeless after uncles who raised him in a Providence, R.I., housing project returned to Puerto Rico.

Davi, 20, lost Social Security disability assistance because the government now considers most H.I.V.-infected people able to work, said Scott Mitchel, a counselor with AIDS Care Ocean State, who got him into an apartment that his agency owns. But Davi has trouble staying employed, following rules, working with managers.

“I don’t think right now he can go out there and support himself,” Mr. Mitchel said. For one thing, his medication, five pills twice daily, causes severe insomnia and diarrhea.

Nowadays, people contracting H.I.V. through sex or drugs may take one easily tolerated pill, but the H.I.V.-baby generation often needs complex multipill doses with irritating side effects, making pill-skipping more likely.

In desperation, doctors sometimes allow them to stop medication altogether rather than take their “last rescue regimen poorly,” Dr. Cooper said.

Davi sometimes skips several days, and “when I feel down, like I just want to give up, I don’t want to take my medicine at all,” he said. “If I didn’t have that kid, I probably never would take them.”

That kid is the son born three years ago after Davi, who was 16, told his girlfriend at the time, who was 14, “I want to have a kid” and “she was cool with it,” he said. “I didn’t really know what I was really doing.”

She took medication during pregnancy, and their son is uninfected. Her family, fearful of Davi’s disease, blocked access to the boy for awhile, and the couple broke up. He is facing assault charges for striking her during an argument, but now sees his son regularly.

He said he wants to stay alive, but “maybe my lifespan is not as long as a normal lifespan. I was born this way and that’s what it is.”

Medication is not the problem for Elizabeth. Eighteen, white, from a wealthy Massachusetts suburb, she has been ostracized and tormented, “ ‘H.I.V. slut’ being yelled across the hallway, anything you can think of,” she said.

Elizabeth did not know she had H.I.V. until the age of 14, when her parents and physician appeared at her therapist’s appointment and told her.

“I couldn’t speak or really breathe,” said Elizabeth, whose mother was infected through a blood transfusion before she was born.

Elizabeth, who asked that her last name be withheld, said her mother “wants me to be completely closed about it,” and even Elizabeth’s little sister, who is uninfected, does not know. Keeping it inside feels “like holding your breath underwater for too long.”

But close friends she confided in betrayed her. Her best friend gossiped about it, and a boyfriend she broke up with “told everyone to get back at me,” she said. Schoolwork suffered as she constantly feared hazing and “focused on having to deal with this.”

At 16, she told a new boyfriend, who “promised that he’d never judge me upon it, that he’d never break up with me, that he’d never tell anyone,” she said. “The next day he broke up with me because of it.” They reunited, but his parents scorned her and he sometimes hid their relationship from friends, said Elizabeth, who recently stopped seeing him.

She said people in her well-educated community, who should know that H.I.V. can be well controlled with medication and protected against with condoms, have been surprisingly intolerant.

“There’s no need to think I’m dirty,” she said. “I’ve basically had my trust for people completely taken away.”

Sharing and Trusting

Things get harder as H.I.V. babies grow up and leave the “very nurturing network” of pediatric AIDS clinics and programs, said Rena Greifinger, who formed the One Love Project to help such young people. “At 18, all that support melts away,” plus some of them “have been completely rejected by their families, the leper child.”

At a weeklong retreat at Babson College in Wellesley, Mass., One Love provided music therapy, role-playing about disclosing H.I.V. status, and explicit discussions about sex and having children.

“When I was pregnant, I was crying all the time, worried she would be positive,” said Imani Walters, 19, who contracted H.I.V. as a teenager. Her daughter turned out healthy. “Then you get scared you might not see them grow up.”

The young people were told that having infected children was unlikely with medication through pregnancy, avoiding breastfeeding and giving the babies medication for six weeks.

“We wanted to give them so much information that we get that buy-in to stay on their meds, and they learn how to live with H.I.V.,” said Bill Kubicek, executive director of Next Step, which sponsors One Love and was co-founded by Paul Newman.

Elizabeth met other H.I.V. babies for the first time at the conference, and “here I trust everyone,” she said.

The gathering was eye-opening for Sandy Perez, 18, from Canaan, N.H., too. Her mother, infected through drug use, died when Sandy was 7. Some foster families mistreated her, she said; at one home, underfed, she slept in the laundry room, locked in, and would climb out the window and re-enter the house through the garage to grab food.

Although she now has a loving family and takes medication regularly, she has experienced serious symptoms: sunken cheeks, gaunt face, bony arms. Medication has caused diabetes, and liver and kidney problems.

Sandy rarely disclosed her H.I.V., not even to boyfriends, although she always used condoms. But she said the conference “inspired me” to feel “comfortable with myself and with having this H.I.V., so I can now share it with people that I feel I trust.”

Like her boyfriend of two years, with whom she had not had intercourse. Unable to reach him by phone from the conference, she texted him: “I have H.I.V.” He texted back: “Are you serious?” She replied: “Yes.”

Sandy felt relieved. If he did not know and “became infected, then there would be a part of me that felt responsible and guilty and just icky inside,” she said.

Soon after, the relationship ended, on good terms, she said. “He kind of didn’t really know how to handle it,” she said. But “he calmed down and we eventually talked” and “he was happy I had told him before we did anything.”

A Family’s Acceptance

When the Cosgroves adopted Tom, he was unruly and angry, and at 5 was further traumatized by seeing his mother “turning different colors, losing her hair,” dying of AIDS, he said. He pasted her obituary on his First Communion banner, and had “nightmares, night sweats,” he said, “very mad at her because I thought she purposefully gave me this disease.”

Since “people were petrified to take any of these children,” Ms. Cosgrove said, she adopted four other “throwaway babies,” saying she was abused as a child and “kind of a throwaway baby myself.”

All four, who arrived untested for H.I.V., turned out negative. “I really felt alone,” Tom said, and at 9, after an H.I.V.-camp friend died, he begged them to adopt someone like him.

They adopted Tyree, who had two H.I.V. strains, one from each parent, and developmental delays. For years, he took medication through a stomach tube because he would vomit it otherwise. Now 11, he often needs leg braces, and his viral load — the amount of virus in the blood — is too high.

Tyree’s adoption did not improve everything for Tom, and Tom was “very out of control” for years, he said. To tame his behavior, Ms. Cosgrove said, she had to “push him down and say, ‘You are not going to talk like that, you are not going to act like that.’ One day I dragged him across the carpet, and his teacher called and I told her this is what I did and why.”

By 12, Tom had calmed down, but he chafed at people’s reactions. Ms. Cosgrove informed his school about his H.I.V. and held a meeting for parents, but while school officials were supportive, some parents and classmates shunned him. Tyree attends another school, whose principal advised against disclosure because parents would react badly. And a soccer coach Ms. Cosgrove told said, “Oh, we’re not taking kids like that.”

Tyree said he feels “lonely” because “none of my friends come over.” He already knows how to protect others, saying, “Don’t have sex with me.”

Tom, now mature and thoughtful, finds things more complicated. With his infection well suppressed with medication, he has started Job Corps, a federal job-training program, but his medications’ side effects affecting his memory concern him, and he said that girlfriends have not lasted long because “people have a lot of worries about going out with someone with this disease.”

Still, since his adoptive family “can accept me for who I am,” he said, “I look at it as if there’s other people out there who can probably do the same.”

    As H.I.V. Babies Come of Age, Problems Linger, NYT, 5.11.2010, http://www.nytimes.com/2010/11/06/us/06hiv.html

CT Scans Cut Lung Cancer Deaths, Study Finds

November 4, 2010
The New York Times
By GARDINER HARRIS

WASHINGTON — Annual CT scans of current and former heavy smokers reduced their risk of death from lung cancer by 20 percent, a huge government-financed study has found. Even more surprising, the scans seem to reduce the risks of death from other causes as well, suggesting that the scans could be catching other illnesses.

The findings represent an enormous advance in cancer detection that could potentially save thousands of lives annually, although at considerable expense. Lung cancer will claim about 157,000 lives this year, more than the deaths from colorectal, breast, pancreatic and prostate cancers combined. Most patients discover their disease too late for treatment, and 85 percent die from it.

No screening method had proved effective at reducing mortality from the disease. Four randomized controlled trials done during the 1970s showed that chest X-rays, while they helped catch cancers at an earlier stage, had no effect on overall death rates. Since then, researchers have suggested that CT scans — which use coordinated X-rays to provide three-dimensional views — could detect lung tumors at an even earlier stage than X-rays.

“This is the first time that we have seen clear evidence of a significant reduction in lung cancer mortality with a screening test in a randomized controlled trial,” said Dr. Christine Berg of the National Cancer Institute.

Cancer doctors and others predicted that the study’s results would soon lead to widespread use of CT scans, in particular for older smokers, who have a one in 10 chance of contracting lung cancer.

“These people are worried about lung cancer, and now there is an opportunity to offer them something,” said Dr. Mary Reid, an associate professor of oncology at the Roswell Park Cancer Institute in Buffalo.

But health officials involved in the study refused to endorse widespread screening of current or former smokers, saying more analysis of the study’s results is needed to further identify who benefited most. Such an analysis is months away. And they pointed out that the study offers no reassurance about the safety of smoking or the advisability of CT scans for younger smokers or nonsmokers.

“No one should come away from this thinking that it’s now safe to continue to smoke,” said Dr. Harold E. Varmus, director of the National Cancer Institute.

Patients wishing to get a CT lung screen will most likely have to pay the roughly $300 charge themselves, since few insurers pay for such scans unless an illness is suspected. The federal Medicare program will soon reconsider paying for such screens, a Medicare official said.

The study, called the National Lung Screening Trial, was conducted by the American College of Radiology Imaging Network and the cancer institute. It involved more than 53,000 people ages 55 to 74 who had smoked at least 30 pack-years — one pack a day for 30 years or two packs a day for 15 years. Ex-smokers who had quit within the previous 15 years were included in the group.

Each was given either a standard chest X-ray or a low-dose CT scan at the start of the trial and then twice more over the next two years. Participants were followed for up to five years. There were 354 lung cancer deaths among those who received CT scans and 442 among those who got X-rays. The $250 million study, which began in 2002, was paid for by the cancer institute and carried out at 33 sites.

Its preliminary results were announced days after an independent monitoring board determined that the benefits of CT scans were strong enough to stop the trial. The study will be published in the coming months.

The study found that for every 300 people who were screened, one person lived who would otherwise have died during the study. But one-quarter of those given CT scans were found to have anomalies, nearly all of which were benign. These false signals generally led to more worry, more CT scans and sometimes to lung biopsies and thoracic surgery.

“There are economic, medical and psychological consequences of finding these abnormalities,” Dr. Varmus said.

Deaths due to all causes declined by 7 percent among study participants who received CT scans, suggesting the tests helped to detect other life-threatening diseases besides lung cancer.

Dr. Claudia Henschke, a clinical professor of radiology at Mount Sinai Medical Center and a longtime advocate for use of CT to screen for lung cancer, said the study was likely to have underestimated the benefits of CT scans because participants were screened only three times. Had the screening continued for 10 years, as many as 80 percent of lung cancer deaths could have been averted, she said. Dr. Henschke’s research has been controversial because of its statistical methods and its financing, which included money from a tobacco company. She earns royalties from makers of CT machines.

“What we also have found is that low-dose CT scan gives information on cardiovascular disease, emphysema” and other pulmonary diseases, Dr. Henschke said. “Those are the three big killers of older people. There is just tremendous potential.”

But Dr. Edward F. Patz Jr., professor of radiology at Duke who helped devise the study, said he was far from convinced that a thorough analysis would show that widespread CT screening would prove beneficial in preventing most lung cancer deaths. Dr. Patz said that the biology of lung cancer has long suggested that the size of cancerous lung tumors tells little about the stage of the disease.

“If we look at this study carefully, we may suggest that there is some benefit in high-risk individuals, but I’m not there yet,” Dr. Patz said.

Since 46 million people in the United States smoke and tens of millions more once smoked, a widespread screening program could cost billions annually. Any further refinement of those most at risk could reduce those costs. Low-dose CT scans expose patients to about the same radiation levels as mammograms. Little is known about how the cumulative risks of years of such scans would balance the benefits.

The study’s results could have both legal and political consequences. Suits against tobacco companies have sought to force cigarette makers to pay for annual CT screens of former smokers. But with the science uncertain, those claims have so far been rebuffed. Congress has diverted some research money to create pilot CT lung screening programs, diversions that may gain momentum now.

Some Obama administration officials argued during the debate on the health care law that patients’ health was often harmed by getting too many tests and procedures that, if reduced, would improve health while reducing costs. This study suggests that, at least in lung cancer, spending more on tests saves lives.

Laurie Fenton, president of the Lung Cancer Alliance, which has lobbied for widespread CT lung screening, said the debate about the advisability of such scans is now over.

“The challenge now shifts from proving the efficacy of the method to developing the proper quality standards, infrastructure and guidelines to bring this needed benefit to those at high risk for the disease — now,” Ms. Fenton said.

But Dr. Peter B. Bach, a pulmonologist at Memorial Sloan-Kettering Cancer Center in New York, said no one should rush out and get a CT scan yet because further analysis will better define whom the screening helped.

“Very soon we’ll have an answer about who should be screened and how frequently,” Dr. Bach said, “but we don’t have that answer today.”

    CT Scans Cut Lung Cancer Deaths, Study Finds, NYT, 4.11.2010, http://www.nytimes.com/2010/11/05/health/research/05cancer.html

New DNA Tests Aimed at Reducing Colon Cancer

October 28, 2010
The New York Times
By NICHOLAS WADE

Two new DNA-based tests, one of them described at a meeting in Philadelphia on Thursday, hold the promise of detecting early — and sharply reducing — colon cancer, a disease that afflicts 150,000 people a year in the United States and costs an estimated $14 billion to treat.

The new tests could help most people avoid colonoscopies, which are routinely prescribed for people over age 50. Instead of screening the entire population, doctors could instead refer people for a colonoscopy only if they had tested positive in one of the DNA tests.

Unlike colonoscopy, in which a seeing tube is threaded up the colon, the DNA tests are noninvasive, so more people would take them. Both tests could be brought to market within two years.

One of the tests, developed by Exact Sciences of Madison, Wis., looks in stool samples for the presence of four altered genes that are diagnostic of colon cancer. The test could catch cancerous and precancerous tumors at an early stage, when they are curable, and allow doctors to remove them promptly.

The other test looks in blood for changes in a single gene, called Septin 9, which is not in the Exact Sciences’ panel of four genes. The test has been developed by Epigenomics AG in Germany.

Both tests would be less expensive than colonoscopy, and potentially more effective. Compliance with colonoscopy is low, since people don’t want to have one, and the overall cost per detection is high because most people are healthy, and even colonoscopy misses many tumors in the upper part of the intestine.

Exact Sciences now plans to enlist several thousand patients in a prospective trial designed to win the Food and Drug Administration’s approval. The trial will be completed in 2012 and the test, if approved, should be available shortly thereafter.

“If widely used, and regularly, this test really does have the opportunity to eliminate colon cancer,” said Dr. David A. Ahlquist, a colon cancer expert at the Mayo Clinic and an adviser to Exact Sciences.

The practical value of the tests depends critically on details like their sensitivity, meaning the proportion of tumors that are detected, and their specificity, meaning how many of the positive results are in fact false alarms.

Exact Sciences reported in July that its test was highly sensitive and specific when applied directly to cells taken from tumors. But in the real world, the tumor DNA must be detected in stool samples, even though almost all the DNA comes from the bacteria of the gut. Just 0.01 percent of the DNA is human, and most of this is normal DNA, not the altered DNA of tumors.

The company reported Thursday at a meeting of the American Association for Cancer Research that in a trial of 1,100 patients, the test had detected 64 percent of adenomas, or polyps, larger than one centimeter in diameter, and 85 percent of cancers, as judged by the colonoscopies also given to the patients.

Dr. Ahlquist said he was very pleased with the new results, especially the 64 percent detection rate for precancerous polyps, since these are best targets for intervention.

Although such a detection rate sounds far from perfect, it can be effective if the test is given on a regular schedule. “The Pap smear detects only 50 percent of cervical cancers, but applied over time it virtually eliminates the disease,” Dr. Ahlquist said.

The specificity of the Exact Sciences test is 88 percent, meaning that 12 percent of the time the patient will be given a false alarm. This may be acceptable, given that the worst that will happen is that the patient will get an unnecessary colonoscopy.

“With stool tests, you need a 90 percent specificity,” said Dr. Bert Vogelstein, a cancer expert at Johns Hopkins University who is also an adviser to the company. “Exact Sciences has gotten close to that. There’s a lot of hope for getting a stool-based test.”

Promising results for the blood-based tests were reported this week at a meeting in Barcelona, Spain. Epigenomics said its test had a sensitivity of 86 percent and a specificity of 93 percent.

Proponents of each test note possible weaknesses of the other. Dr. Achim Plum, a vice president for Epigenomics, said that 30 to 40 percent of people have small polyps, but less than 10 percent of these ever become cancerous. The colon-based test may pick up too many of these, sending far too many people in for colonoscopy.

“The health economics of such a test make no sense,” he said.

But the blood-based test may have a similar problem, since a positive signal could come from cancers anywhere in the body. If the patient is advised to take a whole body imaging scan, more false positives could be generated “so you end up doing more harm than good,” Dr. Vogelstein said.

Dr. Plum said that Epigenomics did not see its test as being necessarily in competition with the Exact Sciences test, because the blood-based test would be good alternative for people put off by stool testing.

    New DNA Tests Aimed at Reducing Colon Cancer, NYT, 28.10.2010, http://www.nytimes.com/2010/10/29/health/29cancer.html

The Age of Alzheimer’s

October 27, 2010
The New York Times
By SANDRA DAY O’CONNOR, STANLEY PRUSINER and KEN DYCHTWALD

OUR government is ignoring what is likely to become the single greatest threat to the health of Americans: Alzheimer’s disease, an illness that is 100 percent incurable and 100 percent fatal. It attacks rich and poor, white-collar and blue, and women and men, without regard to party. A degenerative disease, it steadily robs its victims of memory, judgment and dignity, leaves them unable to care for themselves and destroys their brain and their identity — often depleting their caregivers and families both emotionally and financially.

Starting on Jan. 1, our 79-million-strong baby boom generation will be turning 65 at the rate of one every eight seconds. That means more than 10,000 people per day, or more than four million per year, for the next 19 years facing an increased risk of Alzheimer’s. Although the symptoms of this disease and other forms of dementia seldom appear before middle age, the likelihood of their appearance doubles every five years after age 65. Among people over 85 (the fastest-growing segment of the American population), dementia afflicts one in two. It is estimated that 13.5 million Americans will be stricken with Alzheimer’s by 2050 — up from five million today.

Just as President John F. Kennedy, in 1961, dedicated the United States to landing a man on the moon by the end of the decade, we must now set a goal of stopping Alzheimer’s by 2020. We must deploy sufficient resources, scientific talent and problem-solving technologies to save our collective future.

As things stand today, for each penny the National Institutes of Health spends on Alzheimer’s research, we spend more than $3.50 on caring for people with the condition. This explains why the financial cost of not conducting adequate research is so high. The United States spends $172 billion a year to care for people with Alzheimer’s. By 2020 the cumulative price tag, in current dollars, will be $2 trillion, and by 2050, $20 trillion.

If we could simply postpone the onset of Alzheimer’s disease by five years, a large share of nursing home beds in the United States would empty. And if we could eliminate it, as Jonas Salk wiped out polio with his vaccine, we would greatly expand the potential of all Americans to live long, healthy and productive lives — and save trillions of dollars doing it.

Experience has taught us that we cannot avoid Alzheimer’s disease by having regular medical checkups, by being involved in nourishing relationships or by going to the gym or filling in crossword puzzles. Ronald Reagan suffered the ravages of this disease for a decade despite the support of his loving family, the extraordinary stimulation of his work, his access to the best medical care and his high level of physical fitness. What’s needed are new medicines that attack the causes of the disease directly.

So far, only a handful of medications have been approved by the Food and Drug Administration to treat Alzheimer’s, and these can only slightly and temporarily modify symptoms like forgetfulness, disorientation and confusion. None actually slows the underlying neurodegeneration.

In the mid-1980s, when our country finally made a commitment to fight AIDS, it took roughly 10 years of sustained investment (and about $10 billion) to create the antiretroviral therapies that made AIDS a manageable disease. These medicines also added $1.4 trillion to the American economy. The National Institutes of Health still spend about $3 billion a year on AIDS research, while Alzheimer’s, with five times as many victims, receives a mere $469 million.

Most of the medical researchers who study Alzheimer’s agree on what they have to understand in order to create effective drugs: They must find out how the aberrant proteins associated with the disease develop in the brain. They need to model the progression of the illness so they can pinpoint drug targets. And ultimately they must learn how to get drugs to move safely from the blood into the brain.

A breakthrough is possible by 2020, leading Alzheimer’s scientists agree, with a well-designed and adequately financed national strategic plan. Congress has before it legislation that would raise the annual federal investment in Alzheimer’s research to $2 billion, and require that the president designate an official whose sole job would be to develop and execute a strategy against Alzheimer’s. If lawmakers could pass this legislation in their coming lame-duck session, they would take a serious first step toward meeting the 2020 goal.

Medical science has the capacity to relegate Alzheimer’s to the list of former diseases like typhoid, polio and many childhood cancers. But unless we get to work now, any breakthrough will come too late to benefit the baby boomers. Whether the aging of America turns out to be a triumph or a tragedy will depend on our ability to fight this horrific disease and beat it before it beats us.

Sandra Day O’Connor is a retired associate justice of the Supreme Court. Stanley Prusiner, who received the 1997 Nobel Prize in Medicine, is the director of the Institute for Neurodegenerative Diseases at the University of California, San Francisco. Ken Dychtwald, a psychologist and gerontologist, is the chief executive of a company that consults with businesses about the aging world population.

    The Age of Alzheimer’s, NYT, 27.10.2010, http://www.nytimes.com/2010/10/28/opinion/28oconnor.html

Hormone Therapy Worsens Breast Cancer, Study Finds

October 19, 2010
The New York Times
By DENISE GRADY

Hormone treatment after menopause, already known to increase the risk of breast cancer, also makes it more likely that the cancer will be advanced and deadly, a study finds.

Women who took hormones and developed breast cancer were more likely to have cancerous lymph nodes, a sign of more advanced disease, and were more likely to die from the disease than were breast cancer patients who had never taken hormones.

The increased risks were relatively small and are not fully understood. But previous research has found that hormone treatment can cause delays in diagnosis by increasing breast density, making tumors harder to see on mammograms. Delayed diagnosis may increase the risk of death.

It is also possible that hormones may feed the growth of some breast cancers or the blood vessels that tumors need to grow and spread.

The treatment studied was the most commonly prescribed hormone replacement pill, Prempro, which contains estrogens from horse urine and a synthetic relative of the hormone progesterone.

Many doctors assume that women can safely take hormones for four or five years for menopause symptoms like hot flashes and night sweats, said Dr. Rowan T. Chlebowski, the first author of an article published this week in The Journal of the American Medical Association and an oncologist who treats breast cancer patients at the Harbor-U.C.L.A. Medical Center in Torrance, Calif.

“I don’t think you can say that now,” he said. “I know some people have to take it because they can’t function, but the message now is that you really should try to stop after a year or two.”

Dr. Chlebowski said it was not known whether there is any length of time for which these hormones can be taken without increasing breast cancer risk.

The new information comes from the continuing follow-ups with 12,788 women who were in the Women’s Health Initiative, a major federally financed study that compared women taking hormones with a group taking placebos. The study was halted in 2002, three years ahead of schedule, because researchers found that the hormones were causing small but significant increases in the risk of breast cancer, heart disease, strokes and blood clots in the lungs.

The 2002 study had a huge impact. Before it came out, there was a widespread belief that hormones would reduce women’s risk of heart disease and generally keep them youthful, sexy and healthy. For many, the study shattered that faith.

Six million American women had been taking hormones, but the number quickly fell by about half. The breast cancer rate also soon began to decrease, and many researchers attribute that to the drop in hormone use.

The new report increases the average follow-up time to 11 years from the original 5.6 years. It is the first report from the Women’s Health Initiative that includes death rates from breast cancer related to hormone use.

The researchers found small but significant increases in several harmful effects in women who took the hormones. As the study previously showed, women taking hormones are more likely to develop invasive breast cancer. Their rate of the disease was 0.42 percent per year, compared with 0.34 percent per year in the placebo group.

Among women with breast cancer, those who took hormones were more likely to have cancerous lymph nodes, a sign of more advanced disease — 23.7 percent, versus 16.2 percent in the placebo group.

More women who took hormones died from breast cancer — 0.03 percent per year, versus 0.01 percent per year in the placebo group. That translates to 2.6 deaths per 10,000 women per year among those taking hormones, twice the 1.3 deaths per 10,000 in the placebo group.

Among women who had breast cancer, those who took hormones also had a higher death rate from other causes — 0.05 percent per year, versus 0.03 percent per year. In other words, there were 5.3 versus 3.4 deaths per 10,000 women per year — 1.9 extra deaths in hormone users.

Dr. Chlebowski said that a report last year from the Women’s Health Initiative also found that although hormone treatment did not increase women’s rate of lung cancer, hormone use was linked to a higher death rate among women who had the cancer.

Another author of the new study, Dr. JoAnn E. Manson, a professor of medicine at Harvard Medical School and Brigham and Women’s Hospital, said women should not take the hormones at all unless they really need them, for moderate to severe symptoms like hot flashes and night sweats that disrupt sleep and severely affect their quality of life.

At the same time, she said, the new information should not necessarily alarm women who have taken the hormones, because the new report found only one to two additional breast cancer deaths per 10,000 women per year among those taking hormones.

“The data suggest it is cumulative long-term use,” Dr. Manson said. “Women should avoid long-term use. I think that’s the bottom line.”

She said that women who want treatment should take the lowest possible dose that eases their symptoms.

Noting that many women are taking other hormone formulations in hope of avoiding Prempro’s risks, Dr. Manson said that little was known about the drugs and that more research was urgently needed.

She said more research was also needed to find out if women who took hormones early in menopause had the same risks as those who started the drugs later.

Pfizer, which makes Prempro, issued a statement saying it took the new findings seriously, but questioned the mortality figures.

An editorial accompanying the journal article said that the researchers had probably underestimated the increase in deaths from breast cancer due to hormone therapy, and that “with longer follow-up, the deleterious effect will appear larger,” even though the women are no longer taking the drugs.

The editorial writer, Dr. Peter B. Bach, a physician at Memorial Sloan-Kettering Cancer Center in New York, said that although the increase in cancer in the study might seem small, it becomes large when multiplied out over the population. He also questioned the advice being given to women, to consult their doctors about risks and benefits, and to take the lowest possible doses for the shortest possible time.

“The fallback is that doctors and patients should be deciding this on a one-to-one basis, weighing risks and benefits,” Dr. Bach said in an interview. “How do you do that when you don’t know what the risks are?”

He added, “If you care about preventing this disease and keeping women from suffering and dying from it, then it’s hard to look at these drugs and not have serious concerns about them being used, even for what are intended to be relatively short periods of time.”

This article has been revised to reflect the following correction:

Correction: October 19, 2010

An earlier version of this article incorrectly described Dr. Peter B. Bach, writer of an editorial in The Journal of the American Medical Association. He is a physician at Memorial Sloan-Kettering Cancer Center, but he is not an oncologist.

    Hormone Therapy Worsens Breast Cancer, Study Finds, NYT, 19.10.2010, http://www.nytimes.com/2010/10/20/health/20hormone.html

Supreme Court to Consider Vaccine Case

October 11, 2010
The New York Times
By BARRY MEIER

The safety of vaccines is at the heart of a case expected to be heard on Tuesday by the United States Supreme Court, one that could have implications for hundreds of lawsuits that contend there is a link between vaccines and autism.

At issue is whether a no-fault system established by Congress about 25 years ago to compensate children and others injured by commonly used vaccines should protect manufacturers from virtually all product liability lawsuits. The law was an effort to strike a balance between the need to provide care for those injured by vaccines, some of them severely, and the need to protect manufacturers from undue litigation.

Under the 1986 National Childhood Vaccine Injury Act, such claims typically proceed through an alternative legal system known as “vaccine court.” Under that system, a person is compensated if their injury is among those officially recognized as caused by a vaccine. That person, or their parents, can choose to reject that award and sue the vaccine’s manufacturer, but they then face severe legal hurdles created by law to deter such actions.

The case before the Supreme Court is not related to autism. But the biggest effect of the court’s ruling, lawyers said, will be on hundreds of pending lawsuits that contend a link exists between childhood vaccines and autism. Repeated scientific studies have found no such connection.

Also, in several test case rulings over the last two years, administrative judges in vaccine court have held that autism-related cases did not qualify for compensation. During the last decade, about 5,800 of the 7,900 claims filed in vaccine court, or about 75 percent, have been autism-related, federal data show.

Federal data shows that $154 million was paid in fiscal 2010 to 154 claimants involved in vaccine court proceedings. That figure was significantly higher than in preceding years and reflected several unusually high awards, officials involved in the program said.

In the five preceding fiscal years, an average of $68 million in compensation was paid out on an annual basis, federal data indicates. A compensation fund is financed by an excise tax on vaccinations.

The case to be heard on Tuesday involves an 18-year-old woman, Hannah Bruesewitz, who suffered seizures when she was 6 months old and subsequently suffered developmental problems, her parents say, after receiving a type of D.T.P. vaccine that is no longer sold. The D.T.P. vaccine protects against three potentially deadly childhood diseases: diphtheria; pertussis, which is also known as whooping cough; and tetanus.

Ms. Bruesewitz’s parents have contended in court papers that the vaccine’s manufacturer, which is now a part of Pfizer, knew at the time that their daughter was immunized that there was a safer version of the D.T.P. vaccine but did not produce it. The company rejected that contention

Initially, Ms. Bruesewitz’s parents brought a claim on her behalf to the vaccine court, but the severe injuries that she reportedly suffered were removed from the list of those that qualified for compensation a month before the case was heard. An administrative judge in vaccine court subsequently rejected her claim, so her parents filed a product liability lawsuit against Wyeth, a Pfizer unit that had acquired the vaccine’s manufacturer, Lederle Laboratories.

Lower court judges have ruled that her claims are barred by the federal Vaccine Act. As a result, Ms. Bruesewitz, who lives with her family in Pittsburgh and requires specialized care, has not received any compensation, her father, Russell Bruesewitz, said in a telephone interview.

“The cost of her care is an ongoing burden,” Mr. Bruesewitz said.

The Supreme Court review revolves around the narrow question of whether Congress in passing the Vaccine Act intended to bar lawsuits against vaccine manufacturers based on so-called design defect claims. A vaccine design defect claim essentially asserts that the manufacturer should have sold a different vaccine, which plaintiffs say would have been safer than the one used.

Those filing briefs arguing that Congress intended to permit such lawsuits include the American Association for Justice, a plaintiffs’ lawyers group, and the National Vaccine Information Center, an advocacy group.

Those filing briefs arguing that Congress intended to bar them include the solicitor general of the United States, the Chamber of Commerce and several professional medical groups, including the American Academy of Pediatrics.

James M. Beck, a lawyer in Philadelphia who defends makers of drugs and medical devices, said in a phone interview that a ruling in favor of the Bruesewitzes would allow hundreds of lawsuits asserting a link between vaccines and autism to go forward.

“If these cases go forward, it will make it economically unfeasible for anyone to make vaccines in this country,” said Mr. Beck.

Mr. Bruesewitz said that he and his wife were not opposed to vaccination. Instead, he said they pressed his daughter’s claim because he thought that vaccine producers needed to face the threat of litigation to produce safer medications.

“What we want and are concerned about is to make sure that the safety of vaccines in this country is constantly enhanced,” Mr. Bruesewitz said.

    Supreme Court to Consider Vaccine Case, NYT, 11.10.2010, http://www.nytimes.com/2010/10/12/health/12vaccine.html

New York Asks to Bar Use of Food Stamps to Buy Sodas

October 6, 2010
The New York Times
By ANEMONA HARTOCOLLIS

Mayor Michael R. Bloomberg sought federal permission on Wednesday to bar New York City’s 1.7 million recipients of food stamps from using them to buy soda or other sugared drinks.

The request, made to the United States Department of Agriculture, which finances and sets the rules for the food-stamp program, is part of an aggressive anti-obesity push by the mayor that has also included advertisements, stricter rules on food sold in schools and an unsuccessful attempt to have the state impose a tax on the sugared drinks.

Public health experts greeted Mr. Bloomberg’s proposal cautiously. George Hacker, senior policy adviser for the health promotion project of the Center for Science in the Public Interest, said a more equitable approach might be to use educational campaigns to dissuade food-stamp users from buying sugared drinks.

“The world would be better, I think, if people limited their purchases of sugared beverages,” Mr. Hacker said. “However, there are a great many ethical reasons to consider why one would not want to stigmatize people on food stamps.”

The mayor requested a ban for two years to study whether it would have a positive impact on health and whether a permanent ban would be merited.

“In spite of the great gains we’ve made over the past eight years in making our communities healthier, there are still two areas where we’re losing ground — obesity and diabetes,” the mayor said in a statement. “This initiative will give New York families more money to spend on foods and drinks that provide real nourishment.”

New York State, which administers food stamps locally, signed on to the request, which was received by the Agriculture Department on Wednesday evening.

“We appreciate the state’s interest,” a spokesman, Justin DeJong, said. “We will review and carefully consider the state’s proposal.”

In 2004, the Agriculture Department denied a request by Minnesota to prevent food-stamp recipients from buying junk food. The department said that the plan, which focused on candy and soda, among other foods, was based on questionable merits and would “perpetuate the myth” that food-stamp users made poor shopping decisions.

Congress debated but rejected restricting the purchase of sugared drinks with food stamps as part of a 2008 farm bill, Mr. Hacker said. But this year, the chairman of the House’s Agriculture Committee, Collin Peterson, Democrat of Minnesota, said the House should think about such a ban in its deliberations over the next farm bill.

Mr. Bloomberg and his health commissioner, Dr. Thomas A. Farley, said the ban would help curb the city’s obesity epidemic, which they contend has been fueled by rising soda consumption over the past 30 years.

City statistics released last month showed that nearly 40 percent of public-school children in kindergarten through eighth grade were overweight or obese, and that obesity rates were substantially higher in poor neighborhoods. City studies show that consumption of sugared beverages is consistently higher in those neighborhoods.

Dr. Farley and the state’s health commissioner, Dr. Richard F. Daines, said in an Op-Ed article in The New York Times on Thursday that the ban would not reduce the ability of food-stamp recipients to feed their families. “People would still receive every penny of support they now get, meaning they would have at least as much, if not more, to spend on nutritious food,” Dr. Farley and Dr. Daines wrote. “And they could still purchase soda if they choose — just not with taxpayer dollars.”

The health of New Yorkers, and particularly obesity, is one of the mayor’s signature issues. During his first term in office, Mr. Bloomberg expanded the city’s smoking ban to almost all indoor public places, and he is proposing to expand it to beaches, parks and plazas. New York City has banned trans fats in restaurants and requires restaurants to post calorie counts.

The city’s campaign against sugary drinks has been especially aggressive. This week, it introduced ads showing a man drinking packets of sugar. But its attempt to persuade the State Legislature to impose a tax on the drinks was met with skepticism and opposition from the beverage industry and grocery owners.

Tracey Halliday, a spokeswoman for the American Beverage Association, said of the mayor’s request: “This is just another attempt by government to tell New Yorkers what they should eat and drink.”

The number of New Yorkers qualifying for food stamps has grown more than 35 percent in the past couple of years, mirroring a nationwide trend. And the mayor’s proposal could raise concerns about equity, since it is aimed at one segment of the city, its poorest. When Minnesota sought its ban, welfare rights advocates there accused the state of being patronizing to food-stamp users.

Anticipating such criticism, Dr. Farley and Dr. Daines said that the food-stamp program already prohibited the use of benefits to buy cigarettes, beer, wine, liquor or prepared foods.

The ban would affect beverages with more than 10 calories per 8 ounces, and would exclude fruit juices without added sugar, milk products and milk substitutes. A 12-ounce soda has 150 calories and the equivalent of 10 packets of sugar, according to the health department. City health officials say that drinking 12 ounces of soda a day can make a person gain 15 pounds a year.

Dr. Farley and Dr. Daines said that over the past 30 years, the consumption of soda and other sugary beverages in the United States had more than doubled, paralleling the rise in obesity. They blame that trend for the rising rate of diabetes, which now afflicts one in eight adults in New York City, and is nearly twice as common among poor New Yorkers as among wealthier ones.

Told of Mr. Bloomberg’s request on Wednesday, one food-stamp user, Marangeley Reyes, 24, of Harlem, said the mayor should not dictate what foods she bought. Ms. Reyes had just emerged from a Shop Fair supermarket on Lenox Avenue with a 20-ounce bottle of Orange Crush — she drinks at least one a day. But after giving it some more thought, she said, “I probably shouldn’t be drinking so much soda.”


Joel Stonington and Jack Styczynski contributed reporting.

    New York Asks to Bar Use of Food Stamps to Buy Sodas, NYT, 6.10.2010, http://www.nytimes.com/2010/10/07/nyregion/07stamps.html

Stem Cells in Court, Scientists Fear for Careers

October 5, 2010
The New York Times
By AMY HARMON

Rushing to work at Cincinnati Children’s Hospital Medical Center one recent morning, Jason Spence, 33, grabbed a moment during breakfast to type “stem cells” into Google and click for the last 24 hours of news. It is a routine he has performed daily in the six weeks since a Federal District Court ruling put the future of his research in jeopardy.

“It’s always at the front of my brain when I wake up,” said Dr. Spence, who has spent four years training to turn stem cells derived from human embryos into pancreatic tissue in the hope of helping diabetes patients. “You have this career plan to do all of this research, and the thought that they could just shut it off is pretty nerve-racking.”

Perhaps more than any other field of science, the study of embryonic stem cells has been subject to ethical objections and shaped by political opinion. But only a year after the Obama administration lifted some of the limits imposed by President George W. Bush, a lawsuit challenging the use of public money for the research and a conservative shift in Congress could leave the field more sharply restricted than it has been since its inception a decade ago. At stake are about 1,300 jobs, as well as grants from the National Institutes of Health that this year total more than $200 million and support more than 200 projects.

The turn of events has introduced what researchers say is unprecedented uncertainty to a realm of academic science normally governed by the laws of nature and the rules of peer review.

“We’re used to people telling us, ‘That was a stupid idea, we’re not going to fund it,’ and we turn around and think of a better one,” said James Wells, who heads the laboratory where Dr. Spence has a postdoctoral position. “But there’s nothing we can do about this.”

The stem cells, which are thought to have curative potential for many diseases because they can be turned into any kind of tissue in the human body, can be obtained only by destroying a human embryo, which many Americans believe is the equivalent of a life.

In August, Chief Judge Royce C. Lamberth of Federal District Court for the District of Columbia found that the Obama administration’s policy violates a law barring federal financing for “research in which a human embryo or embryos are destroyed, discarded or knowingly subjected to risk of injury or death,” and issued an injunction blocking federal money for the research.

Since then, the field’s fate has appeared to shift almost weekly as the lawsuit wends its way through the courts. Last week, the government won the right from an appeals court to continue financing the contested research while it appeals the ruling. But there is no telling how the appeals court will ultimately rule, and Judge Lamberth could issue a revised injunction.

Many of the nation’s leading stem cell researchers do not know whether they will receive grants they won years earlier through the standard competition, or whether new projects will even be considered. Junior scientists like Dr. Spence, poised to start their own laboratories, are caught in limbo. Senior scientists like Dr. Wells are torn between pursuing research they believe in and protecting students from staking their job prospects on projects they may never be able to complete.

The legal roller coaster is raising stress levels and reducing productivity, researchers say. Instead of tending to their test tubes, they find themselves guessing how each member of the Supreme Court might vote on the case. They are also watching the midterm Congressional elections with new interest — and with some dismay, since many believe that new legislation will be required for their work to continue.

Under guidelines authorized by both the Bush and Obama administrations, work that leads directly to destroying the embryos cannot be federally financed. The government can, however, support subsequent research on the cell lines created by that process.

Last year, two scientists filed the lawsuit, arguing that the distinction is a false one and that the guidelines on public financing violated the Dickey-Wicker amendment, first passed in 1996 and renewed by Congress every year since.

Moreover, they said, it siphons limited government resources from research on different types of stem cells, which they and other scientists who share a discomfort with embryonic stem cells view as ethically and scientifically superior. For all the hope vested in them, human embryonic stem cells have yet to yield tangible results for patients.

In his ruling, Judge Lamberth agreed that the guidelines violated the 1996 amendment and “threaten the very livelihood” of the plaintiffs.

Embryonic stem cell researchers who stand to lose their federal grants as a result argue that other types of stem cells do not have the same properties, and that all need to be studied regardless to determine which work best. They bristle at the intrusion of judges and politicians into decisions usually addressed by the peer review process, in which experts in a field comment on the merit of an idea and the best get financed.

Yet even some who believe there is a compelling scientific rationale for their research agree that the legal basis for federal financing may be weak. “I was astonished that Congress hadn’t dealt with this,” said Stephen Duncan, a stem cell researcher at the Medical College of Wisconsin, who stands to lose several million dollars in federal grants depending on the dispensation of the case. “It’s like being a little pregnant. You’re either breaking the law or you’re not.”

Mr. Bush, who in 2001 limited federally financed researchers to working on roughly two dozen stem cell lines already in existence, twice vetoed legislation that would have explicitly expressed support for financing the contested research. No such legislation has been introduced under President Obama, but the administration expanded the number of stem cell lines researchers could study.

Advocates of the research now see this as a missed opportunity.

Efforts to rally Congressional support since Judge Lamberth’s ruling have failed to gain momentum among Democrats and moderate Republicans heading into the November elections.

For many, the most recent intrusion of politics into the vaunted scientific meritocracy came as a particular shock because the Obama administration’s new guidelines had only months earlier fallen into place.

“The painful thing is that we are being stopped at a time when the velocity of this field of research, thanks to the new administration, was finally going at maximum speed,” said Ali H. Brivanlou, a professor at Rockefeller University.

Over the last few weeks, embryonic stem cell scientists have sought alternative financing from private foundations, university administrations and state programs. But the National Institutes of Health, which has a $26 billion budget, is by far the source with the deepest pockets for academic scientists.

Some researchers are weighing a switch to the private sector. Others have ordered their students to pay no attention to the news. Others are trying to raise public awareness.

Yi Sun, 45, of the University of California, Los Angeles, has resorted to frequent meditation.

“I would be in trouble without it,” said Dr. Sun, whose stem cell work focuses on an autism disorder called Rett syndrome. Born in China, Dr. Sun said she was now renewing efforts to collaborate with well-financed stem cell biologists there.

    Stem Cells in Court, Scientists Fear for Careers, NYT, 5.10.2010, http://www.nytimes.com/2010/10/06/science/06stem.html

Condom Use Is Highest for Young, Study Finds

October 4, 2010
The New York Times
By RONI CARYN RABIN

A wide-ranging study of Americans’ sexual behavior, based on the largest nationally representative survey since 1992, finds that condom use is becoming the norm for sexually active teenagers.

Indeed, they are more responsible than adults about using condoms, the researchers report in a study coming out on Monday. A vast majority of sexually active 14- to 17-year-olds — 80 percent of boys and 69 percent of girls — said they had used a condom the last time they had intercourse, compared with well under half of adults involved in casual liaisons.

“I think that just as teenagers quickly develop an expectation that they’re going to learn to drive no matter where they live,” said a co-author of the survey, Dr. J. Dennis Fortenberry, a professor of pediatrics at the Indiana University School of Medicine, “there’s the same general widespread sense among contemporary teenagers that as you get to the point where you start thinking about having sex, condoms are going to be part of that decision.”

The new study, the first to include participants as young as 14 and as old as 94, finds that decades after the sexual revolution, the gap between men’s and women’s sexual satisfaction persists.

While most men said they had experienced orgasm the last time they had sex, and 85 percent believed their partner had also, only two-thirds of the women surveyed said they had achieved orgasm the last time they had sex. And a startling number of women — almost one-third — said they had experienced pain the last time they had sex (only 5 percent of men did).

The report is drawn from the National Survey of Sexual Health and Behavior, carried out by researchers at the Center for Sexual Health Promotion at Indiana University. It was based on responses from 5,865 individuals, including about 800 under 18.

The report was welcomed by health providers and sex educators, who say the field has had little data to go on, even as monumental social changes — the gay rights movement, increases in cohabitation, later marriage and childbearing, the AIDS epidemic and the widespread use of drugs for erectile dysfunction, among others — have transformed sexual attitudes.

Government agencies and private foundations are reluctant to pay for studies of sexual behavior that do not focus on reproduction, experts say; the last broad sex survey, the 1992 National Health and Social Life Survey, was started under a governmental contract, but Congress cut off financing and it was completed with support from private sources.

“There’s been an enormous explosion of research in virtually all areas of medicine except the entity called sexual medicine,” said Dr. Irwin Goldstein, editor in chief of The Journal of Sexual Medicine, which is devoting its entire issue to papers from the study and commentaries by experts. “We can’t get the funding.”

Even though Americans are bombarded with sexual imagery, they have little access to reliable information about sexual behavior, said Monica Rodriguez, president of Siecus, the Sexuality Information and Education Council of the United States, a nonprofit group based in New York. “That’s why this is so important — it gives us a sense of what’s really happening, instead of all this, ‘Well, my sex life must not be normal, because I don’t do this or only do this.’ ”

The new study was financed by Church & Dwight, the maker of Trojan condoms. Researchers said that while they had shared information with the sponsor during the course of the study, the company had not exerted influence over the way it was conducted, except to offer advice on how to phrase questions to accurately gauge condom use.

The study also found that while only about 7 percent of men and of women identified themselves as “other than heterosexual,” a much higher percentage reported having had sex with a member of the same sex. Among women in their 30s, for example, 14 percent said they had performed oral sex on another woman at some point in their lives; 13 percent of men over 40 said they had performed oral sex on another man.

    Condom Use Is Highest for Young, Study Finds, NYTY, 4.10.2010, http://www.nytimes.com/2010/10/04/health/04sex.html

Suicides Put Light on Pressures of Gay Teenagers

October 3, 2010
The New York Times
By JESSE McKINLEY

FRESNO, Calif. — When Seth Walsh was in the sixth grade, he turned to his mother one day and told her he had something to say.

“I was folding clothes, and he said, ‘Mom, I’m gay,’ ” said Wendy Walsh, a hairstylist and single mother of four. “I said, ‘O.K., sweetheart, I love you no matter what.’ ”

But last month, Seth went into the backyard of his home in the desert town of Tehachapi, Calif., and hanged himself, apparently unable to bear a relentless barrage of taunting, bullying and other abuse at the hands of his peers. After a little more than a week on life support, he died last Tuesday. He was 13.

The case of Tyler Clementi, the Rutgers University freshman who jumped off the George Washington Bridge after a sexual encounter with another man was broadcast online, has shocked many. But his death is just one of several suicides in recent weeks by young gay teenagers who had been harassed by classmates, both in person and online.

The list includes Billy Lucas, a 15-year-old from Greensburg, Ind., who hanged himself on Sept. 9 after what classmates reportedly called a constant stream of invective against him at school.

Less than two weeks later, Asher Brown, a 13-year-old from the Houston suburbs, shot himself after coming out. He, too, had reported being taunted at his middle school, according to The Houston Chronicle. His family has blamed school officials as failing to take action after they complained, something the school district has denied.

The deaths have set off an impassioned — and sometimes angry — response from gay activists and caught the attention of federal officials, including Secretary of Education Arne Duncan, who on Friday called the suicides “unnecessary tragedies” brought on by “the trauma of being bullied.”

“This is a moment where every one of us — parents, teachers, students, elected officials and all people of conscience — needs to stand up and speak out against intolerance in all its forms,” Mr. Duncan said.

And while suicide by gay teenagers has long been a troubling trend, experts say the stress can be even worse in rural places, where a lack of gay support services — or even openly gay people — can cause a sense of isolation to become unbearable.

“If you’re in the small community, the pressure is hard enough,” said Eliza Byard, executive director of the Gay, Lesbian, and Straight Education Network, which is based in New York. “And goodness knows people get enough signals about ‘how wrong it is to be gay’ without anyone in those communities actually having to say so.”

According to a recent survey conducted by Ms. Byard’s group, nearly 9 of 10 gay, lesbian, transgender or bisexual middle and high school students suffered physical or verbal harassment in 2009, ranging from taunts to outright beatings.

In Mr. Clementi’s case, prosecutors in New Jersey have charged two fellow Rutgers freshmen with invasion of privacy and are looking at the death as a possible hate crime. Prosecutors in Cypress, Tex., where Asher Brown died, said Friday that they would investigate what led to his suicide.

In a pair of blog postings last week, Dan Savage, a sex columnist based in Seattle, assigns the blame to negligent teachers and school administrators, bullying classmates and “hate groups that warp some young minds and torment others.”

“There are accomplices out there,” he wrote Saturday.

In an interview, Mr. Savage, who is gay, said he was particularly irate at religious leaders who used “antigay rhetoric.”

“The problem is that kids are being exposed to this rhetoric, and then they go to the school and there’s this gay kid,” he said. “And how are they going to treat this gay kid who they’ve been told is trying to destroy their family? They’re going to abuse him.”

In late September, Mr. Savage began a project on YouTube called “It Gets Better,” featuring gay adults talking about their experiences with harassment as adolescents.

In one video, a gay man named Cyrus tells of his life as a closeted teenager in a small town in upstate New York.

“The main thing I wanted to come across from this video is how different my life is, how great my life is, and how happy I am in general,” he says.

Glennda Testone, the executive director of the Lesbian, Gay, Bisexual & Transgender Community Center in New York City, said their youth programs serve about 50 young people a day, often suffering from “bullying, harassment or even violence.”

“The three main groups of pivotal figures are family, friends and their schoolmates,” she said. “And if they’re feeling isolated and like they can’t tell those people, it’s going to be a very rough ride.”

Here in Fresno, in California’s conservative Central Valley, groups like Equality California have been more active in trying to establish outreach offices, particularly after an election defeat in 2008, when California voters approved Proposition 8, which banned same-sex marriage.

In Tehachapi, in Kern County south of here, more than 500 mourners attended a memorial on Friday for Seth Walsh. One of those, Jamie Elaine Phillips, a classmate and friend, said Seth had long known he was gay and had been teased for years.

“But this year it got much worse,” Jamie said. “People would say, ‘You should kill yourself,’ ‘You should go away,’ ‘You’re gay, who cares about you?’ ”

Richard L. Swanson, superintendent of the local school district, said his staff had conducted quarterly assemblies on behavior, taught tolerance in the classroom and had “definite discipline procedures that respond to bullying.”

“But these things didn’t prevent Seth’s tragedy,” he said in an e-mail. “Maybe they couldn’t have.”

For her part, Ms. Walsh said she had complained about Seth’s being picked on but did not want to cast blame, though she hoped his death would teach people “not to discriminate, not be prejudiced.”

“I truly hope,” she said, “that people understand that.”


Ian Lovett contributed reporting from Tehachapi, Calif.

    Suicides Put Light on Pressures of Gay Teenagers, NYT, 3.10.2010, http://www.nytimes.com/2010/10/04/us/04suicide.html

Dr. William Harrison, Defender of Abortion Rights, Dies at 75

September 25, 2010
The New York Times
By DOUGLAS MARTIN

“Oh, God, doctor, I was hoping it was cancer.”

Those words so affected Dr. William Harrison that for years, he said, he could not repeat them. They made him break down in tears.

The woman who spoke them — black, poor and middle-aged — had come in 1967 to the Arkansas hospital where Dr. Harrison was a medical student in obstetrics. A doctor, after examining her swollen belly, had told her she was pregnant.

Dr. Harrison went on to perform 20,000 or so abortions over three decades, became a frequent target of abortion protesters and spoke out forcefully in national forums. In the 1980s, when the abortion wars raged fiercest in Arkansas, his clinic, the Fayetteville Women’s Clinic, was firebombed, picketed, blockaded and vandalized. Death threats became routine.

In essays, articles and interviews he castigated abortion-rights opponents as “right-wing crazies.” They responded just as fervently, calling him a murderer, particularly after he gained wide attention for offering free abortions to survivors of Hurricane Katrina in 2005.

Dr. Harrison felt compelled to perform abortions after many doctors in northwest Arkansas abandoned the practice. For decades, he was the only doctor in the region willing to provide the procedure. In typically flamboyant language, he said, “I have chosen to ride this tiger unquietly, raking its side with verbal spurs, swinging my hat and whooping like a cowboy.”

Dr. Harrison died on Friday at the age of 75, the Nelson-Berna Funeral Home in Fayetteville said. He had received a diagnosis of leukemia in May.

Dr. Harrison readily admitted that he destroyed life, but denied that he killed babies. His view was that an embryo was far from being a human being with a brain. The higher moral value to Dr. Harrison was salvaging the future of an often disadvantaged girl or woman.

But he drew a line at performing abortions in the third trimester of pregnancy, partly, he said, because they made him uncomfortable and partly because he felt he lacked the expertise. He sent those who wanted one to his friend Dr. George Tiller in Wichita, Kan., an expert surgeon who was assassinated in his church by an anti-abortion zealot in 2009. Dr. Harrison often gave those patients money for gasoline for the trip to Wichita.

William Floyd Nathaniel Harrison, the son of small-town teachers, was born in rural Faulkner County, Ark., on Sept. 8, 1935. His mother would say that she knew he was destined to become an obstetrician because he essentially delivered himself as his father rode on horseback to fetch the doctor. He grew up going to Methodist and Baptist churches (his mother played piano at both), and twice read the entire Bible at 12, ending up, he said, thoroughly unimpressed with the God it described.

By his account, he piled up D’s and F’s at what is now the University of Central Arkansas in Conway before enlisting in the Navy at 17. He later enrolled at the University of Arkansas, where he studied business until he fell for Betty Waggoner. She was dating a pre-med student, he said, so he switched to that to impress her. They were married for 50 years.

In addition to his wife, Dr. Harrison is survived by his daughters, Amanda Robinson and Rebecca Harrison; his son, Benjamin III; a brother, Ben; two sisters, Mary Harrell and Martha Harrison; and seven grandchildren.

Dr. Harrison received his undergraduate and medical degrees from the University of Arkansas and did his internship and residency there. He and a colleague set up the Fayetteville Women’s Clinic in 1972.

Dr. Harrison performed his first abortion in 1974, a year after they became legal, and found himself doing them sporadically while his clinic was booming delivering babies. But by 1984, as older doctors retired and younger ones shied from offering abortions, often fearing being ostracized or attacked, he became the only doctor in his area performing them.

As more and more abortion patients knocked on his door, he said, he began recalling the woman who would have preferred cancer to pregnancy. He also recalled the many women who had come to the hospital seriously injured by illegal abortions.

So after delivering more than 6,000 babies, he gave up that practice and devoted himself to abortions, writing that if he wanted them to be legal, safe and available, the only moral and ethical course was for him to do them.

As for the protesters frequently outside his clinic, he often said they were splendid advertising, drawing women in need to the clinic who might otherwise have not known where to go.

    Dr. William Harrison, Defender of Abortion Rights, Dies at 75, NYT, 25.9.2010, http://www.nytimes.com/2010/09/26/us/26harrison.html

About Your Premiums

September 24, 2010
The New York Times

With health insurers jacking up their premiums by double-digit amounts for people who buy their own policies, Republicans have been predictably eager to blame health care reform. Consumers, and voters, beware.

If your premiums are spiking suddenly, you can blame economic reality. The cost of medical care continues to soar upward, and the recession led many healthy people to drop coverage, leaving less-healthy enrollees who cost more to insure.

As for health care reform, the major elements, and major costs, don’t even kick in until 2014. The only provisions with the potential to affect premiums right now are a handful of consumer protections that are popular with the public, and not especially costly to implement.

Starting this week, insurers are prohibited from setting lifetime limits and restricted in setting annual limits on what they will pay for care, banned from rescinding policies after a beneficiary becomes sick and prohibited from excluding children with preexisting conditions. The new rules will also allow dependents up to the age of 26 to remain on their parents’ policies; preventive care will be provided without cost sharing; and there are new processes to appeal insurance company decisions. The critics aren’t saying anything about the huge value of those immediate benefits.

These new requirements do impose some new costs on insurers, but they certainly can’t be blamed for the double-digit increases many insurers have been seeking. If you listen closely to the insurers, they admit that.

The Department of Health and Human Services estimates that the average premium increase to pay for the new benefits should be about 1 percent to 2 percent, roughly consistent with an independent evaluation by the Urban Institute. If those estimates are optimistic, they are unlikely to be off by much. A Wall Street Journal analysis found insurers in various states seeking increases of 1 percent to 9 percent to pay for the new requirements and often even more to cover rising medical costs.

Some of the largest health insurers created a stir this week when they announced that they will no longer issue new child-only policies to people who buy their own policies. They were worried that, with no immediate mandate that every child carry insurance, parents would hang back until their children got sick.

It is disappointing but less draconian than it seems.

The market for child-only policies is small, and such children can still be covered through family policies, public programs for low-income people and new high-risk pools. The problem should disappear in 2014 when everyone will be required to carry insurance.

Not all the news about the insurers is bad. As Reed Abelson reported in The Times on Thursday, some companies are preparing to meet the challenge of reform by upgrading information technology systems, training employees and reducing overhead costs. Of course, the critics of health care reform aren’t mentioning that either.

    About Your Premiums, NYT, 24.9.2010, http://www.nytimes.com/2010/09/25/opinion/25sat1.html

F.D.A. to Restrict Avandia, Citing Heart Risk

September 23, 2010
The New York Times
By GARDINER HARRIS

WASHINGTON — In a highly unusual coordinated announcement, drug regulators in Europe and the United States said Thursday that Avandia, the controversial diabetes medicine, would no longer be widely available.

The drug’s sales will be suspended entirely in Europe, while patients in the United States will be allowed access to the medicine only if they and their doctors attest that they have tried every other diabetes medicine and that patients have been made aware of the drug’s substantial risks to the heart. Patients now taking Avandia may continue to do so.

The Food and Drug Administration’s decision shows that the Obama administration is taking a harder line on drug safety issues, even in the face of scientific uncertainty. Along with its announcement, the agency for the first time immediately posted on its Web site internal memorandums from top staff members that in some cases offered entirely contradictory advice. Dr. Margaret Hamburg, the agency’s commissioner, said that passions within the agency had run high on the Avandia decision.

“As F.D.A. commissioner, my job would be infinitely easier if we had consensus and full scientific clarity,” she said.

Dr. Steven Nissen, a Cleveland Clinic cardiologist whose studies highlighted Avandia’s heart attack risks, said that the decision brought an end to “one of the worst drug safety tragedies in our lifetime,” adding that it was “essential to fully investigate what went wrong with the regulatory process to prevent this type of tragedy from endangering patients in the future.”

One study estimated that from 1999 to 2009, more than 47,000 people taking Avandia needlessly suffered a heart attack, stroke or heart failure, or died.

The decision on Avandia signals a new era in the treatment of diabetes, a disease that is reaching epidemic proportions in much of the industrialized world. Because of Avandia, the F.D.A. announced in 2008 that it would no longer approve medicines simply because they help diabetics control blood sugar levels — the standard for more than 80 years. Instead, the F.D.A. now insists that drugmakers conduct trials lasting at least two years to show that their medicines do not hurt the heart and that they improve the quality or length of diabetics’ lives, far tougher tests.

The Avandia story also begins a new and unsettling period for pharmaceutical companies because Avandia’s risks became known only after Dr. Nissen analyzed data from clinical trials that GlaxoSmithKline, the maker of the drug, had been forced to post on its Web site as a result of a legal settlement. Such public postings are increasingly the norm, which means that drugmakers can no longer easily hide or control scientific information about their medicines.

The agency’s decision to order restrictions on Avandia’s sales also demonstrates that the F.D.A. — given new powers over drugmakers and drug distribution in a 2007 law — intends to use those powers. The agency has now ordered that dozens of drugs be sold only with special restrictions.

In explaining why the F.D.A. decided against only adding more warnings to Avandia’s label, Dr. Janet Woodcock, director of the F.D.A.’s drug center said, “We know that labels are often not read.” It was an extraordinary acknowledgment from a veteran of an agency that for decades relied almost exclusively on label warnings to control drug use.

The suspension and restrictions all but ensure that Avandia’s sales — $1.19 billion last year and $3.2 billion as recently as 2006 — will plunge as regulatory authorities around the world are bound to follow with similar restrictions. Avandia was once the biggest-selling diabetes drug around the world, but concerns about heart attack risks have already cut sales. There are about 600,000 people currently taking Avandia in the United States, Dr. Hamburg said at a press conference. “I think the numbers will go down very, very significantly with these new requirements.”

GlaxoSmithKline responded that “the company continues to believe that Avandia is an important treatment for patients with Type 2 diabetes” and is working with the F.D.A. and European regulators “to implement the required actions.” The company promised that it would end Avandia promotions around the world.

The restrictions will take months to put in place. Patients now taking Avandia should continue to do so until they can consult their doctors, said Dr. Joshua Sharfstein, the F.D.A.’s principal deputy commissioner. But he said that doctors should now consider switching patients to other medicines.

European regulators began their own reassessment of Avandia in July after seeing a study of the drug by F.D.A. medical officers and another by Dr. Nissen in advance of a July F.D.A. advisory meeting. Once each agency learned of the other’s impending and similar decision — both were reached independently, Dr. Hamburg emphasized — they decided to coordinate their announcements.

Several consumer groups said that the F.D.A. should have removed Avandia from the market just as the Europeans did.

The F.D.A. announced that it had ordered Glaxo to end a controversial study comparing Avandia and Actos, made by Takeda. It also ordered Glaxo to conduct an independent assessment of the Record trial, a landmark study of Avandia’s heart effects that an F.D.A. medical officer found was riddled with what he said were unpardonable errors that seriously biased the trial’s conclusions.

In 2007, after a study by Dr. Nissen found that Avandia increased the risk of heart attacks, an advisory committee agreed but voted to keep it on the market.

More studies intensified the controversy, so the F.D.A. held another advisory meeting in July. This time, a majority of experts — many of whom had supported Avandia’s continued sales in 2007 — decided that the drug should either be withdrawn or its sales should be severely restricted. With Thursday’s announcements, the F.D.A. and the European Medicines Agency are following that panel’s advice.

Dr. Clifford J. Rosen, a member of the advisory panel that met in July, said he was gratified that the F.D.A. had followed the panel’s advice, and he said the new guidelines “will protect patients while at the same time allowing those few patients who have benefited from treatment to stay on the drug.”

Approved in 1999, Avandia helps control blood sugar levels in diabetics by making patients more sensitive to their own insulin. It is one of a class of three drugs, the first of which, Rezulin, was withdrawn because it caused liver damage. Actos, the last remaining drug in the class, appears safe in part because it seems to affect a different set of genes than either Rezulin or Avandia.

Senate investigators found that GlaxoSmithKline spent years hiding from regulatory authorities clear indications that Avandia increased heart risks. In July, GlaxoSmithKline took a $2.3 billion liability charge related to legal cases involving Avandia and another medicine, Paxil. At the time, investors cheered the news as the company’s attempt to set a ceiling for its liability surrounding the medicine.

But the twin announcements by European and American regulators, along with a string of troubling findings about the company’s conduct by Senate investigators, may increase the company’s legal exposure.

Senator Max Baucus, a Democrat from Montana who helped to spearhead an investigation of Avandia, said he was pleased. “The FDA’s tough new restrictions on the drug will help protect patients,” Mr. Baucus said.

    F.D.A. to Restrict Avandia, Citing Heart Risk, NYT, 23.9.2010, http://www.nytimes.com/2010/09/24/health/policy/24avandia.html

Insurers Scramble to Comply With Health Rules

September 22, 2010
The New York Times
By REED ABELSON

The first big wave of new rules under the federal health care law goes into effect on Thursday, leaving many insurers scrambling to get ahead of the changes.

Insurers are cutting administrative staff to lower overhead costs, investing in big technology upgrades and training employees to field the expected influx of customer inquiries.

Despite the talk among some Republicans of repealing all or part of the law, insurers say they cannot afford to put off the changes. Many said they were fundamentally altering their business models to cope.

“It is really the Manhattan Project because of the scale and the scope,” said Karen Ignagni, chief executive of America’s Health Insurance Plans, a trade group.

Under the new law, insurers that offer child-only policies must start covering all children, even the seriously ill, beginning on Thursday. Insurers must also begin offering free preventive services, and for the first time, their premiums must start passing muster with federal and state regulators by the end of the year.

Companies are choosing to avoid some of the rules by no longer offering certain policies. Aetna, WellPoint and Cigna, for example, have announced that they would stop selling new child-only policies, at least in some states. Ahead of the regulatory review, some also raised premiums this year.

The coming weeks and months should provide an important test of the legislation — and a dry run of sorts for the more far-reaching changes required by the law in 2014, when insurers will have to offer coverage to everyone and begin selling their plans on state-run exchanges.

Adjusting to the new terrain could push some insurers out of business, health care analysts say. In a setback to the bottom line, for example, insurers will no longer be able to pick and choose enrollees to avoid covering people who are likely to run up high medical bills, and many of the markets where they operate will become much less profitable.

“A lot of health plans will struggle and fail,” said Jeff Fusile, a health care partner at PricewaterhouseCoopers.

Blue Shield of California, which was an early proponent of requiring insurers to cover everyone, has trained about 2,500 people — nearly half of its work force — about the impact of the new law. About 250 of the employees are leading teams responsible for reprogramming computer systems, determining the cost of new policies and ensuring that the people answering phones have the correct responses to customer questions, among other tasks.

“The train has left the station, and we’d much rather be the ones with the engineer’s hat,” said Bruce Bodaken, the chief executive of Blue Shield of California, a nonprofit health plan based in San Francisco.

The challenge for the industry as a whole will be to demonstrate an ability to oversee patient care and work closely with hospitals and doctors to find ways to improve the quality of care while trying to contain costs. To that end, insurers are making big investments in technology systems and new areas of expertise.

Blue Shield says it has already increased its efforts to address the cost and quality of care by exploring new ways to pay doctors and hospitals.

“We’re going to do a lot more innovation and experimentation, failing and succeeding, than we’ve ever been doing before,” Mr. Bodaken said.

Like many of its competitors, Blue Shield is also upgrading its technology systems. The plan is spending several hundred million dollars on the effort, and both the new and old systems must be reprogrammed at the same time to reflect the new rules.

But the cost of such upgrades could be the undoing of some plans, said Mr. Fusile, of PricewaterhouseCoopers.

“The biggest challenge facing the payers is that they are going through a pretty massive investment in information technology,” he said.

Even those insurers that were critical of the legislation have little choice but to comply. Still, several have chosen to avoid the new rules by ceasing to offer certain policies. In the case of the child-only policies, insurers say the new rules could require them to cover too many sick children and too few healthy ones.

Aetna, for one, says it would be willing to work with government officials to find ways to keep offering the coverage. “With the right regulatory environment, we would be able to keep plans affordable and continue to offer them,” said Matt Wiggin, an Aetna spokesman.

The Obama administration says that it is willing to work with the insurers, but added that the companies had already promised to continue offering the policies.

“Insurance companies have pledged to offer coverage to children with pre-existing conditions, and we expect them to honor that commitment,” said Jessica Santillo, a spokeswoman for the Department of Health and Human Services.

One of the most critical changes in the coming months will be the final rules dictating how insurers spend their premiums. The federal legislation says that at least 80 cents of every dollar collected in premiums must be spent on the welfare of patients, but the regulations spelling out what qualifies are still being worked out.

Some insurers are acting quickly to make cuts to lower their overhead expenses in anticipation of the new regulations on spending, said Roger Collier, a former health care executive who is now a consultant. Brokers may see their commissions, which are paid for by the insurers, sharply reduced or eliminated as one potential source of savings, he said.

The legislation also requires insurers to have their premiums vetted, in some fashion, by regulators at the state and federal levels. Given the amount of controversy over the double-digit increases some plans have already requested, insurers are readying themselves for a long and potentially fraught process.

Blue Shield recently filed rate increases of less than 2 percent, on average, for all of its policies, and a little more than 4 percent in the individual market, largely to address the expanded benefits under the new federal law.

The timetable to enact the new rules under the health care law is aggressive, and administration officials say they will enforce it. Still, the administration recently announced its willingness to allow insurers more leeway in complying with some rules when the logistical challenges were essentially insurmountable.

The rush to comply has led to some stumbles. When Blue Shield, like some of its competitors, wanted to offer coverage to adult children up to age 26 through their parents’ policies, the insurer notified its customers about the change — only to discover that some employers, who had the final say over whether to speed up the timing of the change, wanted to wait. Blue Shield then sent out follow-up letters delaying the change.

    Insurers Scramble to Comply With Health Rules, NYT, 22.9.2010, http://www.nytimes.com/2010/09/23/business/23insure.html

For Many, Health Care Relief Begins Today

September 22, 2010
The New York Times
By KEVIN SACK

Sometimes lost in the partisan clamor about the new health care law is the profound relief it is expected to bring to hundreds of thousands of Americans who have been stricken first by disease and then by a Darwinian insurance system.

On Thursday, the six-month anniversary of the signing of the Patient Protection and Affordable Care Act, a number of its most central consumer protections take effect, just in time for the midterm elections.

Starting now, insurance companies will no longer be permitted to exclude children because of pre-existing health conditions, which the White House said could enable 72,000 uninsured to gain coverage. Insurers also will be prohibited from imposing lifetime limits on benefits.

The law will now forbid insurers to drop sick and costly customers after discovering technical mistakes on applications. It requires that they offer coverage to children under 26 on their parents’ policies.

It establishes a menu of preventive procedures, like colonoscopies, mammograms and immunizations, that must be covered without co-payments. And it allows consumers who join a new plan to keep their own doctors and to appeal insurance company reimbursement decisions to a third party.

The arrival of the long-awaited changes propelled President Obama, whose Democrats have struggled to exploit their signature achievement, into the backyard of Paul and Frances Brayshaw of Falls Church, Va., to explain his decision to pursue health care.

“The amount of vulnerability that was out there was horrendous,” Mr. Obama on Wednesday told a gathering of people chosen to illustrate the law’s new provisions. He said he concluded that “we’ve just got to give people some basic peace of mind.”

Mr. Obama also responded to Republican Congressional leaders who have campaigned on a threat to repeal the act. “I want them to look you in the eye,” he told his audience, and explain their opposition to a law that is projected to cover 32 million uninsured and reduce the deficit by $143 billion over 10 years.

The Republican strategy “makes sense in terms of politics and polls,” Mr. Obama said, an acknowledgment that the electorate is divided and that many swing districts are hostile. “It just doesn’t make sense in terms of actually making people’s lives better.”

House Republicans continued to question Mr. Obama’s assertions, which he repeated Wednesday, that the law will lower premiums, pointing to double-digit increases recently announced by many insurers. A blog posting on the Web site of the minority leader, Representative John A. Boehner of Ohio, predicted the law would “raise health care costs, explode the federal deficit and create a byzantine bureaucracy.”

The administration has estimated that premiums should rise no more than 2 percent because of the new consumer protections, and warned this month that it would have “zero tolerance” for efforts to blame the law for larger increases.

It will take years to determine the act’s long-term impact on American health, and on American politics. But Democrats did manage to front-load some notable benefits, while deferring the pain of tax increases and penalties until after the election.

Polls have found that many of the provisions taking effect Thursday are popular, tugging at a national sense of fairness and feeding off distrust of health insurers. They bear particular appeal for the 14 million people who must buy policies on the individual market rather than through employers and are thus at the mercy of the industry. And they land on the heels of a government report showing that the recession drove the number of uninsured Americans to 50.7 million in 2009, up 10 percent in a year.

As the political battle endures, those most immediately affected are welcoming the changes with collective relief, and hoping that their promise of security is real.

    For Many, Health Care Relief Begins Today, NYT, 22.9.2010, http://www.nytimes.com/2010/09/23/health/policy/23careintro.html

Benefits and Burdens of Medicaid

September 21, 2010
The New York Times

The vital importance and the staggering cost of Medicaid have become painfully clear over the last two years. Millions of out-of-work Americans have turned to Medicaid to help pay their medical bills. State governments, which share the cost with Washington, have had to struggle to find the money; many have cut back on other vital programs to try to balance their budgets.

Consider New York State, which currently covers more than 4.5 million poor residents. Of this year’s projected $52.6 billion Medicaid price tag, the federal government will pick up $31.1 billion. The state will pay $14.2 billion and local governments another $7.3 billion.

Federal stimulus funds that have been easing the strain will expire in June. The new health care reform law will expand Medicaid enrollments in 2014 and will increase the state’s costs, even with enhanced federal support. That means New York and all states have to rein in Medicaid spending. In a new report, Lt. Gov. Richard Ravitch offers some sensible recommendations.

Short of cutting eligibility and benefits, which Mr. Ravitch is rightly not proposing, the states have limited tools, so long as health care costs continue to soar. The next governor should consider Mr. Ravitch’s ideas.

New York’s Medicaid program is administered by several state agencies and 58 local governments. Mr. Ravitch would give the state director more control to ensure tighter, more efficient management. He also would take the power to set reimbursement formulas for hospitals, doctors and other health care providers away from the Legislature and give it to the state Medicaid director. The director would be advised by an expert panel, similar to one that has long advised the federal government on Medicare reimbursements, with modest success.

Mr. Ravitch also highlights the need for a comprehensive strategy to finance long-term care for the chronically ill, which accounts for almost half of New York’s Medicaid spending. Whether the solution is greater reliance on managed care organizations, as he suggests, or some other coordinated care would have to be debated.

Mr. Ravitch has other longer-term ideas. He would establish an innovation center (analogous to a federal office created by health care reform) to develop new ways to deliver and pay for health care; increase support for preventive care; and reform medical malpractice. That is a worthy objective, but more study is needed to determine how capping awards harms patients.

Mr. Ravitch is also urging the state’s political leaders to push for changes in Medicaid financing formulas so New York and other states with large numbers of poor get more help from Washington. That is long overdue. Mr. Ravitch is right that New York and all states need to start thinking hard about what they can do to make Medicaid more efficient, while also ensuring that millions of vulnerable people have access to care.

    Benefits and Burdens of Medicaid, 21.9.2010, http://www.nytimes.com/2010/09/22/opinion/22wed2.html

New Drugs Stir Debate on Basic Rules of Clinical Trials

September 18, 2010
The New York Times
By AMY HARMON

Growing up in California’s rural Central Valley, the two cousins spent summers racing dirt bikes and Christmases at their grandmother’s on the coast. Endowed with a similar brash charm, they bought each other matching hardhats and sought iron-working jobs together. They shared a love for the rush that comes with hanging steel at dizzying heights, and a knack for collecting speeding tickets.

And when, last year, each learned that a lethal skin cancer called melanoma was spreading rapidly through his body, the young men found themselves with the shared chance of benefiting from a recent medical breakthrough.

Only months before, a new drug had shown that it could safely slow the cancer’s progress in certain patients. Both cousins had the type of tumor almost sure to respond to it. And major cancer centers, including the University of California, Los Angeles, were enrolling patients for the last, crucial test that regulators required to consider approving it for sale.

“Dude, you have to get on these superpills,” Thomas McLaughlin, then 24, whose melanoma was diagnosed first, urged his cousin, Brandon Ryan. Mr. McLaughlin’s tumors had stopped growing after two months of taking the pills.

But when Mr. Ryan, 22, was admitted to the trial in May, he was assigned by a computer lottery to what is known as the control arm. Instead of the pills, he was to get infusions of the chemotherapy drug that has been the notoriously ineffective recourse in treating melanoma for 30 years.

Even if it became clear that the chemotherapy could not hold back the tumors advancing into his lungs, liver and, most painfully, his spine, he would not be allowed to switch, lest it muddy the trial’s results.

“I’m very sorry,” Dr. Bartosz Chmielowski, the U.C.L.A. oncologist treating both cousins, told Mr. Ryan’s mother, Jan. He sounded so miserable that afternoon that Mrs. Ryan, distraught, remembers pausing to feel sorry for the doctor.

Controlled trials have for decades been considered essential for proving a drug’s value before it can go to market. But the continuing trial of the melanoma drug, PLX4032, has ignited an anguished debate among oncologists about whether a controlled trial that measures a drug’s impact on extending life is still the best method for evaluating hundreds of genetically targeted cancer drugs being developed.

Defenders of controlled trials say they are crucial in determining whether a drug really does extend life more than competing treatments. Without the hard proof the trials can provide, doctors are left to prescribe unsubstantiated hope — and an overstretched health care system is left to pay for it. In melanoma, in particular, no drug that looked promising in early trials had ever turned out to prolong lives.

PLX4032 shrinks tumors in the right patients, for a limited time. But would those who took it live longer? No one knew for sure.

“I think we have to prove it,” said Dr. Paul B. Chapman, a medical oncologist at Memorial Sloan-Kettering Cancer Center who is leading the trial. “I think we have to show that we’re actually helping people in the long run.”

But critics of the trials argue that the new science behind the drugs has eclipsed the old rules — and ethics — of testing them. They say that in some cases, drugs under development, PLX4032 among them, may be so much more effective than their predecessors that putting half the potential beneficiaries into a control group, and delaying access to the drug to thousands of other patients, causes needless suffering.

“With chemotherapy, you’re subjecting patients to a toxic treatment, and the response rates are much lower, so it’s important to answer ‘Are you really helping the patient?’ ” said Dr. Charles L. Sawyers, chairman of human oncology at Sloan-Kettering. “But with these drugs that have minimal side effects and dramatic response rates, where we understand the biology, I wonder, why do we have to be so rigorous? This could be one of those defining cases that says, ‘Look, our system has to change.’ ”

Dr. Richard Pazdur, director of the cancer drug office at the Food and Drug Administration, said in a recent interview that the new wave of drugs in development — especially for intractable cancers like melanoma — might require individual evaluation. “This is an unprecedented situation that will, hopefully, be increasingly common, and it may require a regulatory flexibility and an open public discussion,” he said.

And doctors say that for them, the new wave of cancer drugs is intensifying the conflict between their responsibility to their patients and their commitment to gathering scientific knowledge for generations of the critically ill.

Of course, no single pair of patients can fairly represent the outcomes of a trial whose results are not yet known. Rather, the story of Thomas McLaughlin and Brandon Ryan is one of entwined paths that suddenly diverged, with a roll of the dice.

At times beseeching and belligerent, Mr. McLaughlin argued his cousin’s case to get the new drug with anyone he could find at U.C.L.A. “Hey, put him on it, he needs it,” he pleaded. And then: “Who the hell is making these decisions?”

He believed he should trade places on the trial with Mr. Ryan, who was pursuing his contractor’s license and had just bought a four-bedroom home in Bakersfield. “Brandon has everything going for him,” he told his Aunt Jan.

But Mr. Ryan told his mother he was glad that Mr. McLaughlin, who has a young son and daughter, was the one getting the promising drug. “Tommy has the kids,” he said. “They need him around.”

Path to a Second Trial

The debate over the controlled testing of PLX4032 began in June 2009, around the time Mr. McLaughlin awakened with what felt like an explosion under his right armpit.

The drug, manufactured by Roche, the Swiss pharmaceutical giant, was designed for melanoma patients whose tumors carry a particular mutation, and the company reported that month that nearly all 32 such patients in the drug’s first clinical trial, called Phase 1, had seen their tumors shrink.

The reprieve was all too brief: most saw their tumors begin to grow again within the year. Still, The New England Journal of Medicine called the drug “a major breakthrough” for people with advanced melanoma, whose median survival is eight months after diagnosis. A second, or Phase 2, trial, aiming to validate the results in more patients, was already in the works. And in meetings that summer, several oncologists urged Roche to seek accelerated approval from the F.D.A. The agency allows a manufacturer to sell a drug based on early promise so long as it proceeds with the traditional controlled trial comparing it with the standard treatment.

But with patients already begging doctors for the drug, it seemed unlikely that anyone would join a trial with only a 50-50 chance of getting PLX4032 once it was already on the market. Unless the trial was conducted before approval, it seemed, there would be no chance to get definitive data on its effectiveness.

Some melanoma specialists familiar with the drug would have traded the data for faster access to the drug. “I know all that I need to know based on the results we already have,” said Dr. Keith Flaherty of Massachusetts General Hospital, who led the early clinical testing. “My use of this drug is not going to be informed by testing it against a drug we all hate and would rather never give a dose of again in our lives.”

The standard chemotherapy used in melanoma, dacarbazine, slowed tumor growth in 15 percent of patients for an average of two months. By contrast, PLX4032 had halted tumor growth in 81 percent of patients for an average of eight.

It was conceivable that when the cancer started up again, it would progress much faster in patients who had taken the new drug, wiping out any extra time they might have gained. But even if so, many doctors believed that if the drug provided relief by shrinking tumors — like the one Mr. McLaughlin soon learned was pressing against a nerve in his arm — that would improve their patients’ lives.

The trial, moreover, would cost $100 million and delay the possibility of F.D.A. approval by at least two years. To some doctors, it seemed a waste of time and resources that would be better used for trials testing what everyone most cared about: how to prolong the remissions.

There was reason to believe that combining PLX4032 with other drugs — some from competitors — would make it more effective. But researchers had to rely on Roche for permission until the drug was available for sale, and the company had not been forthcoming.

Dr. Chapman of Sloan-Kettering came up with a new tack: an unconventional bid to speed the drug’s approval, rooted in the observation that patients weeks or days from death could get out of bed and off oxygen when given PLX4032, sometimes for months. The doctors working with the drug referred to this as the Lazarus effect; it was unheard of with dacarbazine.

A trial that cataloged PLX4032’s effect on the well-being of the sickest patients, Dr. Chapman argued, would probably yield fast, tangible results. For him, it represented a chance to give patients symptomatic relief, even if the drug turned out not to prolong life.

“Even without a survival benefit, maybe we could show that it helps people,” he urged. “If you could get Aunt Sadie to the wedding and off of oxygen, that would be great.”

But company officials feared that might lead to approval for only a narrow group of the sickest patients. The surest way to get the F.D.A’s endorsement for a broader market was a controlled trial. And with its competitors rushing to get similar drugs to market, the findings of such a trial might give Roche an advantage in marketing its version as the only one proven to prolong survival.

On Sept. 1 last year, the company submitted its plan to the F.D.A. for the traditional, randomized, controlled trial of PLX4032. It would involve 680 patients, half of them in a control group. Dr. Chapman would be the lead investigator for more than 100 sites in the United States, Europe and Australia. Because of the different ways the drugs were dispensed — one by mouth and one by infusions — doctors and patients, it was decided, would both know who got which drug.

The following week was when Mr. Ryan learned that his cousin might have a health problem. He called Mr. McLaughlin from a job site in Colorado, to tell him about his new Dodge Ram, a truck he knew Mr. McLaughlin had long coveted.

He invited Mr. McLaughlin to come stay with him: there was plenty of welding work, and he could help break in the truck. But Mr. McLaughlin, who had no health insurance, had finally visited a doctor about the pain under his arm. It was melanoma, and he would need surgery to remove some lymph nodes.

“Wow,” Mr. Ryan said, suddenly silent. “You have cancer?”

Two Men’s Struggles

Mr. McLaughlin’s surgery, it seemed, had come too late. In the weeks following, small tumors popped up across his body, including one on his collarbone and one on his triceps.

When Mr. Ryan discovered a swollen node under his own right armpit in October, his mother was not taking any chances. She begged him to go to the emergency room in Colorado. Even so, when the verdict was melanoma, both families were shocked.

Was it genes? Their mothers, after all, were sisters. But there was no history of cancer in the family.

Environment? The boys had fought, played and competed with each other since childhood: who could hold his breath the longest, do the highest cannonball dive, suck down a Slurpee fastest, win their grandfather’s approval? They had ranged across California on iron-working jobs, eating the same food, drinking the same large quantities of beer, promising, in a rare moment of seriousness, that each would bury the other with his hardhat when the moment came. Coincidence?

Compared with most cancers, melanoma strikes a disproportionate number of young people; it is the sixth most common cancer in the United States.

There was no way to know.

Last Thanksgiving, Mr. McLaughlin greeted Mr. Ryan with the usual bear hug. “Looks like we’re doing this together,” he said.

Not ones for excessively talking things over, they left it at that.

Yet both cousins, like the other family members, believed then that Mr. Ryan stood a far better chance of surviving the disease than his cousin. His cancer was rated Stage 3, with no evidence yet that it had spread to distant parts of his body. Mr. McLaughlin, at Stage 4, had a tumor ominously near his liver. And Mr. Ryan had health insurance, while Mr. McLaughlin had none.

It was the mutated gene that the U.C.L.A doctor found in Mr. McLaughlin’s cancer cells in December that turned his luck around. Called B-RAF, it goes awry in half of the 68,000 Americans who develop melanoma each year, for reasons not well understood, signaling cells to grow uncontrollably.

The mutation meant that he would be eligible for PLX4032’s new trial, so the cost of the drug and doctors’ visits would be paid by Roche. And it turned out he would get the pills even before the controlled study began, on a small test of the drug’s interaction with common drugs like caffeine and cough syrup. Judging by the response of patients to PLX4032 in the first trial, Mr. McLaughlin was almost certain to respond. But the medication, the doctors at U.C.L.A warned him, might cause a rash and fatigue and would probably make his skin extremely sensitive to the sun.

“They told me to get a job where I could be inside all the time,” Mr. McLaughlin told Mr. Ryan with a grin; perhaps no one else could better understand how ridiculous it seemed for someone who had spent his whole life outdoors.

Because the slots in the trial were reserved for patients with the most advanced cancer, Mr. Ryan was not eligible — yet. But because he had few symptoms, it hardly seemed to matter. After surgery to remove his cancerous lymph nodes and radiation, he was preparing to return to work.

“Dude, I had ALL of my lymph nodes out,” Mr. Ryan boasted to his cousin over a Mexican-style Christmas dinner at their grandmother’s home in Santa Maria, not passing up an opportunity at one-upmanship. “How many did you have out again, 11?”

Mr. McLaughlin, fingering the tumor that felt like a knot under his arm, might not have been in top form that evening. But he mustered a scoffing reply: “So you had all of them taken out and only four had tumors?”

The following week, he took his first pills.

But even as the tumor on Mr. McLaughlin’s collarbone began to melt away, a faint spot on Mr. Ryan’s lung began to grow.

A Life-or-Death Debate

The discontent among some oncologists over the design of the PLX4032 trial spilled over at a scientific meeting sponsored by the Melanoma Research Alliance in late February.

The ethical review boards at dozens of prestigious cancer research institutions had signed off on the trial, and the leading melanoma oncologists had embraced it: after all, it was the only way to get the most promising drug available for their patients.

But with the trial now under way, a few attending the Las Vegas meeting had already had to tell patients they had been assigned to the trial’s chemotherapy control group. And some had begun to question whether an ethical code that calls for doctors to be genuinely uncertain about which of a trial’s treatments will be more effective had been breached when it came to PLX4032 versus dacarbazine.

After Dr. Chapman presented the recent data from the drug’s promising first trial to a packed room, Dr. Neal Rosen, a friend and Sloan-Kettering colleague, stood up.

“Excuse me,” Dr. Rosen said with unusual formality. “But if it was your life on the line, Doctor, would you take dacarbazine?”

The room was silent.

“My goal,” Dr. Chapman shot back, “is to find out as quickly as possible in as few patients as possible whether this works. If we never know, then we’re never going to be able to build on anything.”

One of the melanoma field’s senior clinicians, Dr. Chapman had lived through trial after trial of drugs that failed to live up to early promise. Almost every oncologist knew, too, of a case nearly 20 years earlier when bone marrow transplants appeared so effective that breast cancer patients demanded their immediate approval, only to learn through a controlled trial that the transplants were less effective than chemotherapy and in some cases caused death.

“Making patients’ tumors go away is gratifying,” Dr. Chapman told critics. “But that’s not the business I’m in. I’m in the business of making people live longer. That’s what I want to do.”

Several of the most veteran melanoma doctors agreed with him. But others argued that oncologists had an ethical obligation to push both the F.D.A. and Roche to make the drug more immediately available.

Some of the strongest criticism came from laboratory researchers who study the biology of the disease and see the drug as fundamentally different from its predecessors. The previous red herrings, they argued, never had such a high response rate. Few other drugs had shrunk tumors in as high a percentage of patients with melanoma or any other solid tumor as PLX4032 had in its first human trial.

“Many of my colleagues who are outstanding clinical investigators have been able to convince themselves that this is a fair thing to do,” Dr. David E. Fisher, a leading melanoma biologist at Massachusetts General, said of the controlled trial. “My personal view is it’s nuts. I don’t know anyone who hasn’t shuddered at the concept that we can’t let patients on the control arm cross over because we need them to die earlier to prove this point.”

In the meantime, some doctors were searching for other trials that could help patients worsening in the chemotherapy group of the Roche trial, even at the risk of undermining its results. Several lobbied to get such patients slots on a new trial of a PLX4032 competitor, manufactured by GlaxoSmithKline.

“It’s much easier to tell patients, ‘We’ll try this for six weeks; if it’s working, great, if not, we’ll shift you right away to the other trial,” said Dr. Jeffrey A. Sosman of the Vanderbilt-Ingram Cancer Center in Nashville. “That’s how I’m going to be able to live with the randomization.”

The reason to prevent patients in the chemotherapy group from subsequently getting PLX4032 was to ensure a clean comparison. But who could prevent them from trying treatments that might well help them live longer? At least one melanoma patient left Sloan-Kettering’s care to join the Glaxo trial at New York University.

In April, Mr. McLaughlin donned a bandanna, a sun hat, a long-sleeved shirt and pants and went to a job building fences on a nearby ranch. The pills, he had vowed, would not prevent him from working outside.

Mr. Ryan’s health, by contrast, was declining. He returned from work only to sleep. Often, when his mother called, he was too tired to come to the phone. “Sleeping, Mom,” he would text her. Or “You have no idea what this feels like, Mom.” Or just, “I hurt.”

His doctor in Bakersfield moved up a scheduled scan.

At the same time, a debate grew heated over Roche’s decision to withhold PLX4032 from many patients not eligible for the trial because they had already been treated with chemotherapy.

The F.D.A. regularly approves such programs, known as “compassionate use,” for promising experimental drugs. But Roche feared a prospective trial candidate might undergo chemotherapy just to qualify for compassionate use and get PLX4032 with no strings attached.

In an emotional moment, Dr. Donald Lawrence of Massachusetts General Hospital e-mailed colleagues about Roche’s decision last spring, under the subject line “moral outrage.”

“Just had yet another conversation with a [patient] with a B-RAF mutation who will die in the next month or so because he can’t get PLX4032,” he wrote. “I feel we need to muster the support of our patients and lobby both Roche and the F.D.A. Compromising the Phase III trial is not justification for withholding an effective drug from dying patients.”

But Dr. Michael Atkins, director of the cancer clinical trials office at Beth Israel Deaconess Cancer Center in Boston, urged him to consider what he thought was the greater good: “Even though it is painful, I think completing a clean Phase III trial and determining if there truly is a survival benefit for PLX would have major value for the field and future patients.”

A Bitter Blow

On the morning of May 12, Mr. Ryan and his mother drove to U.C.L.A. The cancer had spread throughout his body. Yet that weekend, the family was filled with hope. Dr. Chmielowski had found the same gene mutation that Mr. McLaughlin had in one of Mr. Ryan’s tumors. He was finally eligible for the trial.

But the computer made its assignment the following Tuesday, making sure that he would not be getting his cousin’s “superpills.”

Mr. Ryan’s mother picked up the call while her son was undergoing radiation for the tumor on his spine. He was on oxygen.

“I’m sorry,” Dr. Chmielowski repeated as she cried into the phone.

There must be someone higher up to whom she could talk, she said.

There was not, he told her. It was completely random. No one could change it.

“Who else has this drug?” Mrs. Ryan demanded. “We will go wherever we have to go.”

There was nowhere to go, the doctor explained. Once Mr. Ryan had been randomly assigned to the control group at one place, the other hospitals testing the melanoma drug would not give it to him. U.C.L.A. had turned away such patients, too.

The doctor did not tell Mrs. Ryan about the Lazarus effect — that for someone as sick as Mr. Ryan, PLX4032 was probably the best chance to control his symptoms while doctors searched for something better.

The doctor could not know, of course, whether Mr. Ryan really would have fared better on the Roche drug, or whether Mr. McLaughlin’s disease would have been held in check just as well with the chemotherapy. Obeying the trial’s protocol meant withholding the drug from patients like Mr. Ryan, and that, Dr. Chmielowski would later explain, “is awful.”

He told Mrs. Ryan, if the chemotherapy could stabilize her son for just a month or so, there were two new trials opening that might help him.

“What gives them the right to play God?” Mrs. Ryan exploded at home later that night. “It doesn’t make sense to say, ‘We want you for a statistic’ instead of giving them a chance at life.”

Mr. Ryan started his infusion the next day. But a week later, he was hospitalized, unable to breathe on his own and in horrible pain.

“Bud brownies,” Mr. McLaughlin prescribed when he arrived to visit, having already signed himself up for medical marijuana use. “You get out of here, and I’ll make them for you.”

He rated the nurses, trying to make Mr. Ryan laugh.

“Maybe you should just say you want to split some of your pills with her and she’ll hop into bed with you,” he suggested after one left the room. A few minutes later, “No, that one’s a little cuter.”

Then he reminded his cousin of the time Mr. Ryan had thrown a bolt up to where he was sitting atop a wall for a welding job adjacent to a golf course. Mr. Ryan missed his mark by several feet and the bolt landed on the other side, shattering the windshield of another contractor’s truck.

“I’m like, ‘You just tagged that guy’s freakin’ truck,’ ” Mr. McLaughlin recounted for the other family members in the hospital room. On his side of the wall, Mr. Ryan had picked up a stray golf ball. “And then the guy walks out and Brandon goes, ‘Looks like those golfers hit your windshield.’ ”

In his hospital bed, Mr. Ryan was beginning to smile.

“And the guy gets in the truck,” Mr. McLaughlin finished, “and takes off for the golf course.”

Two weeks later, at his cousin’s funeral in mid-June, Mr. McLaughlin placed Mr. Ryan’s hardhat in his coffin and helped carry it to the grave.

Mr. McLaughlin has now been taking PLX4032 for nine months. He is awaiting his next CT scan.

    New Drugs Stir Debate on Basic Rules of Clinical Trials, NYT, 18.9.2010, http://www.nytimes.com/2010/09/19/health/research/19trial.html

Precursor to H.I.V. Was in Monkeys for Millennia

September 16, 2010
The New York Times
By DONALD G. McNEIL Jr.

In a discovery that sheds new light on the history of AIDS, scientists have found evidence that the ancestor to the virus that causes the disease has been in monkeys and apes for at least 32,000 years — not just a few hundred years, as had been previously thought.

That means humans have presumably been exposed many times to S.I.V., the simian immunodeficiency virus, because people have been hunting monkeys for millenniums, risking infection every time they butcher one for food.

And that assumption in turn complicates a question that has bedeviled AIDS scientists for years: What happened in Africa in the early 20th century that let a mild monkey disease move into humans, mutate to become highly transmissible and then explode into one of history’s great killers, one that has claimed 25 million lives so far?

Among the theories different researchers have put forward are the growth of African cities and the proliferation of cheap syringes.

Confirming that the virus is very old also helps explain why it infects almost all African monkeys but does not sicken them. Over many generations, as any disease kills off vulnerable victims, the host adapts to it.

The new research, published Thursday in Science magazine, was relatively simple. Scientists tested 79 monkeys from Bioko, a volcanic island 19 miles off the West African coast. Bioko used to be the end of a peninsula attached to the mainland in what is now Cameroon, but it was cut off when sea levels rose 10,000 years ago at the end of the last ice age.

Since then, six monkey species have developed in isolation on the island, and scientists from the National Primate Research Center at Tulane University in Louisiana and other American and African universities found that four of them — drills, red-eared guenons, Preuss’s guenons and black colobuses — had members that were infected with S.I.V.

The four strains in the four species were genetically very different from one another — meaning they presumably did not come from monkeys carried over to the island by humans in the last few centuries. But each was close to the strain infecting members of the same four genuses on the mainland, meaning they must have existed before Bioko was cut off.

Knowing that all four strains were at least 10,000 years old, scientists recalculated the virus’s “molecular clock,” measuring how fast it mutates. They now believe that all the S.I.V. strains infecting monkeys and apes across Africa diverged from a common ancestor between 32,000 and 78,000 years ago.

“When we only had 25 years of data, we were dating from the tip at the end of a branch of the evolutionary tree,” said Preston A. Marx, a virologist at the Tulane primate center and an author of the paper in Science. “I knew that what we had before couldn’t be right, because the virus had spread from the Atlantic to the Indian Ocean to the southern end of the continent, and it couldn’t have done that in a couple of hundred years.”

Beatrice H. Hahn, a virologist from the University of Alabama at Birmingham and a discoverer of the simian virus, called the study “a very nice paper,” adding, “This is what people like us have been looking for.”

Previous methods of dating the virus had concluded it was a few hundred to 2,000 years old, “and that just didn’t seem right,” Dr. Hahn said.

The ancestor virus — which, like many diseases, may have crossed into simians from another, still-unknown species — may have existed for millions of years.

That theory was given greater credence two years ago with the discovery that some Madagascar lemurs have in their genomes the remnants of a virus that was not an S.I.V., but related to it. Madagascar, a Texas-size island 250 miles off the southeastern African coast, separated from Africa 160 million years ago. It has no monkeys, but lemurs’ ancestors arrived there, possibly on floating mats of vegetation, probably more than 10 million years ago.

H.I.V., which is almost universally fatal to humans, is obviously very new to us. As Dr. Marx pointed out, if it had been in humans before the 20th century, it would have arrived in the Americas in some of the 12 million Africans kidnapped for the slave trade.

Its immediate ancestor is probably also relatively new to chimpanzees. Last year, Dr. Hahn showed that it can sicken and kill chimps, although not as quickly, meaning they have probably been adapting to it for generations.

The virus has probably crossed over from simians into humans at least five times. There are two human immunodeficiency viruses, H.I.V.-1, by far the most common, and H.I.V.-2, which is milder and rarely seen outside West Africa, and which jumped to humans from sooty mangabeys, a monkey that West Africans hunt and eat.

H.I.V.-1, in turn, has four substrains, designated M, N, O and P. The first, which has spread around the world, clearly came from chimpanzees, as did N and O. But P appears to have crossed over from a gorilla; it was discovered only last year, and in only one woman, who was from Cameroon, where lowland gorillas are hunted for meat.

It is very likely, scientists said, that a little infected monkey or ape blood got into human veins many times in history as hunters cut themselves while butchering carcasses. But even if it sickened those hunters, it probably died out with them or their immediate contacts.

The earliest confirmed H.I.V. case in humans was found in blood drawn in 1959 from a man in Kinshasa, in what was then called the Belgian Congo.

Sometime between the 1800s and 1959, something presumably allowed a human infection with a chimpanzee virus to spread widely enough to evolve into modern H.I.V.-1, which could spread easily among humans.

Dr. Marx believes that the crucial event was the introduction into Africa of millions of inexpensive, mass-produced syringes in the 1950s. Campaigns to wipe out yaws, syphilis, malaria, smallpox and polio required syringes, and many were reused, often with official approval. Traditional healers adopted them for injecting their decoctions, and they became status symbols; a study in Uganda in the 1960s found that 80 percent of families owned one.

Not everyone agrees. Michael Worobey, a virologist at the University of Arizona and another author of the Science paper, said backdating the molecular clock, which he did by comparing the 1959 blood sample with the only other known early one — a paraffin-embedded lymph node from 1960, also from Kinshasa — suggested that the virus emerged closer to 1910, when syringes were handmade, expensive and rare.

He and Dr. Hahn suspect that the growth of colonial cities is to blame. Before 1910, no Central African town had more than 10,000 people. But urban migration rose, increasing sexual contacts and leading to red-light districts.

    Precursor to H.I.V. Was in Monkeys for Millennia, NYT, 16.9.2010, http://www.nytimes.com/2010/09/17/health/17aids.html

Gauging Psychiatric Drugs for Children

September 11, 2010
The New York Times

To the Editor:

“Child’s Ordeal Reveals Risks of Psychiatric Drugs in Young” (front page, Sept. 2) illustrates a disastrous course for one young child. Millions of children with psychiatric and learning disorders get the wrong treatment or no treatment because of a lack of access to proper care, general misinformation about child development, and a lack of training for pediatricians.

The rule of thumb in all medicine is: diagnosis drives treatment. Physicians cannot treat childhood psychiatric disorders if they aren’t trained to diagnose them. Their diagnoses must be correct, and then, just as important, the treatments they provide must be based on the most current science.

In addition, parents must be educated to ask doctors questions about the benefits and risks of psychiatric medications — as well as non-medication treatments — before a child’s treatment begins. We have a public health crisis and a mental health care system in need of transformation.

Harold S. Koplewicz
New York, Sept. 2, 2010

The writer is a child and adolescent psychiatrist and president of the Child Mind Institute.

•

To the Editor:

Child psychiatric disorders are very real illnesses with significant and lasting consequences. Fortunately, they are also quite treatable. Medication can be a helpful and important component of treatment, but medication alone is rarely an adequate or sufficient intervention. It should be used only as part of a comprehensive plan, individualized to the needs of the child and the family.

As your article demonstrates, finding appropriate psychiatric care for young children can be a daunting challenge. In the process, parents need to become effective and persistent advocates. They should find a mental health professional they like and trust, and learn as much as possible about the full range of treatment options. And they should always feel free to get a second opinion. These are not easy diagnoses to make, and no one has all the answers.

David Fassler
Burlington, Vt., Sept. 2, 2010

The writer, a child and adolescent psychiatrist, is a clinical professor of psychiatry at the University of Vermont.

•

To the Editor:

As a child psychologist in private practice for 20 years, I see an alarming increase in the use of medications for young children that is of real concern. I strongly believe that the key to helping most children with psychological, emotional and behavioral problems is educating and empowering the children, parents and schools with tools and strategies to best help facilitate their emotional and social development.

Not only is there a real risk of serious side effects when young children are overmedicated, but also medication often sedates the child enough so that there is no need seen by the family or the school to remediate their deficits. In effect we are medicating the symptoms but not addressing the etiology.

The great tragedy here is that the overuse of medication often prevents these kids from getting the help that they really need in learning how to compensate for their deficits, difficulties and struggles.

Tamar Z. Kahane
Englewood, N.J., Sept. 2, 2010

•

To the Editor:

This excellent article failed to emphasize one critical point. The true motive for “diagnosing” severe medical disorders in thousands of very young children is reimbursement from insurance. No diagnosis, no payment.

Our health care system is founded on rewarding doctors for identifying and documenting pathology. The more pathology, the more money. To extend help to a child and his family for reduced social circumstances and immature parenting is not reimbursable and is therefore often the last resort.

Virginia Byrnes
Boston, Sept. 2, 2010

The writer is a pediatrician.

    Gauging Psychiatric Drugs for Children, NYT, 11.9.2010, http://www.nytimes.com/2010/09/12/opinion/l12drug.html

Before Giving Up Hope on Alzheimer’s

September 6, 2010
The New York Times

To the Editor:

Re “Years Later, No Magic Bullet Against Alzheimer’s” (front page, Aug. 29), about the National Institutes of Health panel that weighed the evidence on this disease:

Readers might conclude that all preventive strategies are futile against Alzheimer’s. However, the N.I.H. panel reported that we do not know with certainty that these measures will work. This is not that same as stating that such measures do not work.

Policy-making and research demand an exceptionally high standard for scientific evidence. Yet it would be foolish for people hoping to prevent or delay this disease to disregard the epidemiological evidence indicating that some measures may be protective.

While spending money on expensive unproven supplements may be unadvisable, people should be encouraged to maintain a healthy diet and to exercise. Known strategies could be validated in future studies.

Martin Aryee
Baltimore, Aug. 29, 2010

The writer is an instructor in the Biostatistics Division, department of oncology at Johns Hopkins University School of Medicine.

•

To the Editor:

Moderate alcohol intake was not mentioned in your article, though the scientific literature includes dozens of epidemiological studies of older adults that compare risks of cognitive decline, including Alzheimer’s disease, for nondrinkers and those who drink moderately.

Two independent meta-analyses of these studies concluded that moderate drinkers have a reduced risk compared with abstainers. Although alcohol abuse is a serious worldwide public health problem, and even light drinking can be detrimental in certain situations, one wonders why no reference was made to this potentially beneficial edge of a double-edged sword.

Michael A. Collins
Edward J. Neafsey
Maywood, Ill., Aug. 31, 2010

The writers are professors of molecular pharmacology at Loyola University Chicago Stritch School of Medicine.

•

To the Editor:

Until we find a cure for Alzheimer’s disease, we should focus on safety and quality of life.

As a neuropsychologist specializing in dementia, I believe that quality management of all dementias includes an individualized assessment of changes in memory, judgment, mood, behavior and any other symptoms that might appear.

We in the trenches of dementia care have a repertory of strategies to help individuals and their families. Good safety at home can, in many cases, delay or prevent a move to a facility.

Rebecca Goodman
Santa Barbara, Calif., Aug. 29, 2010

•

To the Editor:

We agree that there is no strong evidence that aerobic exercise can lower the risk of Alzheimer’s disease. However, there is strong evidence that strokes, even if small, will make dementia worse at any stage of Alzheimer’s.

That means that the accepted guidelines for lowering the risk factors for vascular disease — exercise and drugs that reduce cholesterol and blood pressure — are highly relevant for optimizing brain health at later ages, with or without Alzheimer’s. Helena Chui
Caleb E. Finch
Los Angeles, Aug. 30, 2010

The writers are professors of gerontology at the University of Southern California.

    Before Giving Up Hope on Alzheimer’s, NYT, 6.9.2010, http://www.nytimes.com/2010/09/07/opinion/l07alzheimer.html

F.D.A. Backtracks and Returns Drug to Market

September 3, 2010
The New York Times
By GARDINER HARRIS

Two weeks ago, the Food and Drug Administration announced that it would remove the drug midodrine from the market because the drug’s maker never confirmed that the medicine — approved in 1996 under an abbreviated process — actually worked against dizziness and fainting.

But 100,000 patients take midodrine for conditions many say would otherwise be disabling, and many flooded the agency with complaints. So on Friday morning, top F.D.A. officials announced that they had backtracked and would continue to allow midodrine to be sold. The announcement came after inquiries by The New York Times.

“In a different situation, we might act differently,” said Dr. Joshua Sharfstein, principal deputy commissioner. “But in this case, it does not make sense to pull access to the drug while we get better data.”

Dr. Janet Woodcock, director of the agency’s drug center agreed. “Patients are out there thinking doom is about to fall, and they can’t go about their normal business,” she said.

Lindsey Mills, a 20-year-old from Baltimore, was among those granted a reprieve. Five years ago, she came back from a trip to the mall and found that she could barely stand because of dizziness. After seeing a parade of doctors, a specialist finally prescribed midodrine, which “was the only thing that allowed me to keep some function and attend classes and not be completely bedridden,” Ms. Mills said.

The agency’s flip-flop demonstrates the difficult choices regulators face in policing the nation’s drug market. Cracking down on drug makers sometimes means stranding desperate patients. And now that Congress has given the Food and Drug Administration greater powers to insist on better information about life-saving medicines, such disputes may become more common.

In a similar case, the F.D.A. is now considering whether to withdraw its approval for the use of Avastin, a cancer medicine made by Genentech, for breast cancer. Recent clinical trials failed to prove that Avastin was helpful in treating breast cancer, but patients and some in Congress expressed outrage that the agency could withdraw approval for the use, an action that could lead insurers to stop paying for it.

Midodrine treats a condition known as postural orthostatic tachycardia syndrome, or Pots. Such patients suffer a severe drop in blood pressure when they stand because blood pools in their legs when vessels fail to constrict. Symptoms range from dizziness to fainting, and the condition is most common among teenage girls.

But midodrine’s maker got the drug approved in 1996 under a process known as accelerated approval, in which drugs for severe or life-threatening conditions are approved even though the evidence for their effectiveness is scant. Under the program, drug makers must promise to conduct more rigorous studies to confirm that the medicines work as hoped.

Accelerated approval had its beginnings in the AIDS crisis, when dying patients said they were willing to accept uncertainty in exchange for faster access to possible cures. But more than a third of the 90 drugs approved under the program since 1992 never had studies done proving efficacy. In some cases, drugmakers profited handsomely from sales, refused to invest in promised studies, and bet that the Food and Drug Administration would not risk angering patients by taking action.

The agency’s failure to crack down led to fierce criticism on Capitol Hill, and midodrine became a telling example of these failures. The drug was developed by Roberts Pharmaceutical and originally called ProAmatine. It has yielded more than $257 million in sales, according to government investigators. Roberts was bought in 2000 by Shire Pharmaceuticals. Removing midodrine from the market would have been the first time the F.D.A. took such action under the accelerated program.

Matthew Cabrey, a Shire spokesman, said he could not explain why Roberts never undertook the needed clinical trial. In 2004, Shire conducted a test in which 24 Pots patients were given either midodrine or a placebo, raised to a vertical position and asked to report when they felt like passing out. The trial failed because it was poorly designed, Mr. Cabrey said.

By then, generic versions of the medicine had cannibalized most of Shire’s sales, so the company did not conduct another trial. On Thursday, Mr. Cabrey said that Shire would not appeal the F.D.A.’s decision to remove midodrine from the market since the company now has less than 1 percent of midodrine’s sales. On Friday, Dr. Jeffrey Jonas, Shire’s senior vice president of research and development, said the company had changed its stance and would appeal.

“There is substantial evidence the drug does work,” Dr. Jonas said.

Parisa Emam, 21, said she was just starting her freshman year at Western Washington University in Bellingham when she began fainting and her vision became so blurry that she could no longer read. She had to use a wheelchair until starting on midodrine. Now she can cook and go grocery shopping, she said.

“This fall I’m going to attempt to take three classes, if I can still get hold of the medication,” she said.

    F.D.A. Backtracks and Returns Drug to Market, NYT, 3.9.2010, http://www.nytimes.com/2010/09/04/health/policy/04fda.html

Child’s Ordeal Shows Risks of Psychosis Drugs for Young

September 1, 2010
The New York Times
By DUFF WILSON

OPELOUSAS, La. — At 18 months, Kyle Warren started taking a daily antipsychotic drug on the orders of a pediatrician trying to quell the boy’s severe temper tantrums.

Thus began a troubled toddler’s journey from one doctor to another, from one diagnosis to another, involving even more drugs. Autism, bipolar disorder, hyperactivity, insomnia, oppositional defiant disorder. The boy’s daily pill regimen multiplied: the antipsychotic Risperdal, the antidepressant Prozac, two sleeping medicines and one for attention-deficit disorder. All by the time he was 3.

He was sedated, drooling and overweight from the side effects of the antipsychotic medicine. Although his mother, Brandy Warren, had been at her “wit’s end” when she resorted to the drug treatment, she began to worry about Kyle’s altered personality. “All I had was a medicated little boy,” Ms. Warren said. “I didn’t have my son. It’s like, you’d look into his eyes and you would just see just blankness.”

Today, 6-year-old Kyle is in his fourth week of first grade, scoring high marks on his first tests. He is rambunctious and much thinner. Weaned off the drugs through a program affiliated with Tulane University that is aimed at helping low-income families whose children have mental health problems, Kyle now laughs easily and teases his family.

Ms. Warren and Kyle’s new doctors point to his remarkable progress — and a more common diagnosis for children of attention-deficit hyperactivity disorder — as proof that he should have never been prescribed such powerful drugs in the first place.

Kyle now takes one drug, Vyvanse, for his attention deficit. His mother shared his medical records to help document a public glimpse into a trend that some psychiatric experts say they are finding increasingly worrisome: ready prescription-writing by doctors of more potent drugs to treat extremely young children, even infants, whose conditions rarely require such measures.

More than 500,000 children and adolescents in America are now taking antipsychotic drugs, according to a September 2009 report by the Food and Drug Administration. Their use is growing not only among older teenagers, when schizophrenia is believed to emerge, but also among tens of thousands of preschoolers.

A Columbia University study recently found a doubling of the rate of prescribing antipsychotic drugs for privately insured 2- to 5-year-olds from 2000 to 2007. Only 40 percent of them had received a proper mental health assessment, violating practice standards from the American Academy of Child and Adolescent Psychiatry.

“There are too many children getting on too many of these drugs too soon,” Dr. Mark Olfson, professor of clinical psychiatry and lead researcher in the government-financed study, said.

Such radical treatments are indeed needed, some doctors and experts say, to help young children with severe problems stay safe and in school or day care. In 2006, the F.D.A. did approve treating children as young as 5 with Risperdal if they had autistic disorder and aggressive behavior, self-injury tendencies, tantrums or severe mood swings. Two other drugs, Seroquel from AstraZeneca and Abilify from Bristol-Myers Squibb, are permitted for youths 10 or older with bipolar disorder.

But many doctors say prescribing them for younger and younger children may pose grave risks to development of both their fast-growing brains and their bodies. Doctors can legally prescribe them for off-label use, including in preschoolers, even though research has not shown them to be safe or effective for children. Boys are far more likely to be medicated than girls.

Dr. Ben Vitiello, chief of child and adolescent treatment and preventive research at the National Institute of Mental Health, says conditions in young children are extremely difficult to diagnose properly because of their emotional variability. “This is a recent phenomenon, in large part driven by the misperception that these agents are safe and well tolerated,” he said.

Even the most reluctant prescribers encounter a marketing juggernaut that has made antipsychotics the nation’s top-selling class of drugs by revenue, $14.6 billion last year, with prominent promotions aimed at treating children. In the waiting room of Kyle’s original child psychiatrist, children played with Legos stamped with the word Risperdal, made by Johnson & Johnson. It has since lost its patent on the drug and stopped handing out the toys.

Greg Panico, a company spokesman, said the Legos were not intended for children to play with — only as a promotional item.

Cheaper to Medicate

Dr. Lawrence L. Greenhill, president of the American Academy of Child and Adolescent Psychiatry, concerned about the lack of research, has recommended a national registry to track preschoolers on antipsychotic drugs for the next 10 years. “Psychotherapy is the key to the treatment of preschool children with severe mental disorders, and antipsychotics are adjunctive therapy — not the other way around,” he said.

But it is cheaper to medicate children than to pay for family counseling, a fact highlighted by a Rutgers University study last year that found children from low-income families, like Kyle, were four times as likely as the privately insured to receive antipsychotic medicines.

Texas Medicaid data obtained by The New York Times showed a record $96 million was spent last year on antipsychotic drugs for teenagers and children — including three unidentified infants who were given the drugs before their first birthdays.

In addition, foster care children seem to be medicated more often, prompting a Senate panel in June to ask the Government Accountability Office to investigate such practices.

In the last few years, doctors’ concerns have led some states, like Florida and California, to put in place restrictions on doctors who want to prescribe antipsychotics for young children, requiring a second opinion or prior approval, especially for those on Medicaid. Some states now report prescriptions are declining as a result.

A study released in July by 16 state Medicaid medical directors, which once had the working title “Too Many, Too Much, Too Young,” recommended that more states require second opinions, outside consultation or other methods to assure proper prescriptions. The F.D.A. has also strengthened warnings about using some of these drugs in treating children.

No Medical Reason

Kyle was rescued from his medicated state through a therapy program called Early Childhood Supports and Services, established in Louisiana through a confluence of like-minded child psychiatrists at Tulane, Louisiana State University and the state. It surrounds troubled children and their parents with social and mental health support services.

Dr. Mary Margaret Gleason, a professor of pediatrics and child psychiatry at Tulane who treated Kyle from ages 3 to 5 as he was weaned off the heavy medications, said there was no valid medical reason to give antipsychotic drugs to the boy, or virtually any other 2-year-old. “It’s disturbing,” she said.

Dr. Gleason says Kyle’s current status proves he probably never had bipolar disorder, autism or psychosis. His doctors now say Kyle’s tantrums arose from family turmoil and language delays, not any of the diagnoses used to justify antipsychotics.

“I will never, ever let my children be put on these drugs again,” said Ms. Warren, 28, choking back tears. “I didn’t realize what I was doing.”

Dr. Edgardo R. Concepcion, the first child psychiatrist to treat Kyle, said he believed the drugs could help bipolar disorder in little children. “It’s not easy to do this and prescribe this heavy medication,” he said in an interview. “But when they come to me, I have no choice. I have to help this family, this mother. I have no choice.”

Ms. Warren conceded that she resorted to medicating Kyle because she was unprepared for parenthood at age 22, living in difficult circumstances, sometimes distracted. “It was complicated,” she said. “Very tense.”

Behavior Problems

Kyle was a healthy baby physically, but he was afraid of some things. He spent hours lining up toys. When upset, he screamed, threw objects, even hit his head on the wall or floor — not uncommon for toddlers, but frightening.

“I’d bring him to the doctor and the doctor would say, ‘You just need to discipline him,’ ” Ms. Warren said. “How can you discipline a 6-month-old?”

When Kyle’s behavior worsened after his brother was born, Ms. Warren turned to a pediatrician, Dr. Martin J. deGravelle.

“Within five minutes of sitting with him, he looked at me and said, ‘He has autism, there’s no doubt about it,’ ” Ms. Warren said.

Dr. deGravelle’s clinic notes say Kyle was hyperactive, prone to tantrums, spoke only three words and “does not interact well with strangers.”

He prescribed Risperdal. At the time, Risperdal was approved by the F.D.A. only for adults with schizophrenia or acute manic episodes. The following year it was approved for certain children, 5 and older, with autism and extremely aggressive behavior. It has never been approved by the F.D.A. for use in children younger than 5, although doctors may legally prescribe it as an off-label use.

“Kyle at the time was very aggressive and easily agitated, so you try to find medication that can make him more easily controlled, because you can’t reason with an 18-month-old,” Dr. deGravelle said in a telephone interview. But Kyle was not autistic — according to several later evaluations, including one that Dr. deGravelle arranged with a neurologist. Kyle did not have the autistic child’s core deficit of social interaction, Dr. Gleason said. Instead, he craved more positive attention from his mother.

“He had trouble communicating,” Dr. Gleason said. “He didn’t have people to listen to him.”

After the neurologist review, the diagnosis changed to “oppositional defiant disorder” and the Risperdal continued.

“Yes, I did ask for it,” Ms. Warren said. “But I was at my wit’s end, and I didn’t know what else to do.”

Dr. deGravelle referred her to Dr. Concepcion, who in turn diagnosed Kyle’s condition as bipolar disorder.

“Some children, when they come to me, the parents are really so frustrated,” Dr. Concepcion said in a phone interview. “Especially the mothers are so scared or desperate in getting help. Their children are really acting psychotic.”

Dr. Concepcion also spoke with Dr. Charles H. Zeanah, a Tulane medical professor, who disagreed with both the diagnosis and the treatment. “I have never seen a preschool child with bipolar disorder in 30 years as a child psychiatrist specializing in early childhood mental health,” Dr. Zeanah said.

More Pills

“It’s a controversial diagnosis, I agree with that,” said Dr. Concepcion. “But if you will commit yourself in giving these children these medicines, you have to have a diagnosis that supports your treatment plan. You can’t just give a nondiagnosis and give them the atypical antipsychotic.”

He also prescribed four more pills.

Kyle’s third birthday photo shows a pink-cheeked boy who had ballooned to 49 pounds. Obesity and diabetes are childhood risks of antipsychotics. Kyle smiles at the camera. He is sedated.

“His shell was there, but he wasn’t there,” Ms. Warren said. “And I didn’t like that.”

Dr. Concepcion referred Kyle to the early childhood support program, which has helped about 3,000 preschoolers from low-income families at risk for mental health problems since 2002.

His speech improved. He threw fewer tantrums. “They started working with us as a family,” said Ms. Warren, who also received parenting advice. “That helps.”

Kyle’s treatment was directed by Dr. Gleason, a Columbia medical graduate who had led a team that wrote 2007 practice guidelines for psychopharmacological treatment of very young children.

“Families sometimes feel the need for a quick fix,” Dr. Gleason said. “That’s often the prescription pad. But I’m concerned that when a child sees someone who prescribes but doesn’t do therapy, they’re closing the door that can make longer-lasting change.”

Off most drugs, Kyle started losing weight and his behavior improved. Ms. Warren’s life also improved. She met a man and they moved into their own house five miles out of Opalousas, a town of 25,000. They were married last Saturday.

At their home recently, Kyle and his brother, Jade, ran and played while their baby sister watched from a playpen. Their clothes were neatly folded in a shared bedroom. They often responded “Yes, ma’am” or “Yes, sir.”

“They’re respectful, but they’re hyper kids,” Ms. Warren said. “Once he came off the medication, he’s Kyle. He’s an intelligent person. He’s loud. He’s funny. He’s smart. He’s bouncy. I mean, there’s never a dull moment. He has a few little behavior issues. But he’s like any other normal 6-year-old.”

Kyle paused to show a reading report card from the end of his kindergarten year, with an A grade.

“Awesome job, Kyle!” his kindergarten teacher wrote.

    Child’s Ordeal Shows Risks of Psychosis Drugs for Young, NYT, 1.9.2010, http://www.nytimes.com/2010/09/02/business/02kids.html