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FDA intends to give once-banned thalidomide a 2nd chance against disease

SHARE FDA intends to give once-banned thalidomide a 2nd chance against disease

There was a time when Vicki Walton was so sick she wished she were dead. A bacterial infection consumed her, pushing her fever to 106 degrees. Painful ulcers lined her throat and genitals. Her joints were swollen, her brain inflamed. Her head pounded with unrelenting migraines. Eating was a faint memory; walking, a distant dream.

These were the hideous symptoms of Behcet's Disease, a rare disorder that kept the 30-year-old Wyoming woman confined to her wheelchair at best, bedridden at worst. "Every ounce of me hurt," she recalled. "I seriously thought about Dr. Kevorkian."Then Walton learned of an experimental drug that was working wonders for Behcet's. She began taking two capsules a day in December 1992. The ulcers cleared up practically overnight, and six months later, she was out of her wheelchair. Today, she does volunteer work and serves on the Kemmerer City Council in her home town.

There has been just one problem: The drug is thalidomide, the world's most notorious cause of birth defects. And Vicki Walton is a fertile woman.

Although it has been banned around the globe for the past 35 years, ever since it shocked the world when women who took it in pregnancy gave birth to thousands of infants with missing or stunted limbs, thalidomide is experiencing a resurgence, showing promise for maladies as diverse as cancer, blindness and AIDS. Yet the medicine that Walton calls "my miracle drug" carries heavy baggage; the mere utterance of the word "thalidomide" evokes such horrible memories among doctors that for some women, getting it has been next to impossible.

It took Walton two years to persuade a doctor to help her get thalidomide through a tightly controlled "compassionate use" program run by the Food and Drug Administration. Now, with the FDA announcing it intends to give thalidomide a second chance, approving it for use 37 years after the drug was first rejected, Walton's story offers a cautionary tale.

Should it receive the agency's imprimatur, thalidomide will be the most regulated drug in the nation's history. Under a proposal submitted by Celgene Corp., the company in Warren, N.J. that is seeking to market the drug, women who take thalidomide would be required to undergo monthly pregnancy tests and use two forms of birth control. There would be no automatic refills, and pharmacists would have to register to dispense the drug.

The idea, according to both the FDA and Celgene, is to strike a delicate balance, imposing enough controls to prevent future tragedies, but not enough to prevent women from getting a medicine that could prolong or even save their lives.

Yet so far, very few women have been given access to thalidomide. Of the 1,200 people who have received the drug in recent years through the government's compassionate use program and experimental clinical trials, the FDA estimates, just 100 have been female.

At the same time, some doctors - even those who are testing the drug in government-sponsored studies that require women to be included - express a deep reluctance to give thalidomide to women, in part for fear of creating more deformed babies, in part for fear of being sued.

When the drug eventually reaches the market, doctors will be forced to confront a haunting question: To whom, ultimately, am I responsible? My female patients? Their unborn children? Myself?

"It's an ethical dilemma for doctors," said Dr. Norman Fost, who directs the medical ethics program at the University of Wisconsin and has written extensively on thalidomide. "I think doctors divide on how much risk they are willing to expose future children to."

As Dr. Jeffrey Jacobson, who heads the AIDS program at Mount Sinai Medical Center in New York and has tested thalidomide, said: "There is no clean argument either way."

Some doctors will take no risks at all. Dr. Jeffrey Fessel of San Francisco refused to enroll women in his portion of a thalidomide study, sponsored by the National Institutes of Health, that found the drug is highly effective in treating severe mouth and genital lesions, called apthous ulcers, common to AIDS patients.

"A female physician from the FDA called me and harangued me as to why I'm refusing females the benefits of this important drug," said Fessel, who directs the HIV research unit at Kaiser Permanente Medical Center. "I said, `You want to give this drug to women - YOU give it to women. I'm not going to give it to women. It's as simple as that."

The FDA doctor was Debra Birnkrant, chairwoman of the agency's thalidomide working group. She said her hands were tied. "He was extremely reluctant, so what do you do?," she asked. "You can't force them to include women."

This hesitance is not news to the people at Celgene, where marketing research has found that doctors are extremely cautious about thalidomide. "Doctors will respond that there is a need for drugs to treat these conditions," said Bruce Williams, the company's vice president for marketing. "But when they learn that it's thalidomide, they say, `Jeez, I don't think I would treat any of my female patients with it.' "

That infuriates women's health advocates, who have fought for years for women to have the same access to medical treatments and experimental drugs as men.

"Obviously we don't want to hand these pills out to anybody," said Cynthia Pearson, executive director of National Women's Health Network, a Washington, D.C., advocacy group. "But it's not fair for one single health professional to decide that a woman with a life-threatening disease can't even have the option to consider taking a drug that causes birth defects."