BETTY ANN BOWSER: For 35 years Giselle Cole has been figuring out how to do things most people take for granted. For example, getting to work for her means a car with $12,000 of modifications.
GISELLE COLE: I've got fingers. I've got a bit of a wrist and a bit of an elbow. I've got no ball and socket in my shoulder. We're not amputees. I'm ambulatory.
BETTY ANN BOWSER: Cole also uses modifications at work, where she is in charge of lingerie distribution for a major retail corporation in Toronto. She has shortened arms and only three fingers on each hand because her mother took a drug called thalidomide while she was pregnant with Giselle. At the time, thalidomide was widely prescribed for morning sickness in countries all over the world. What no one knew until babies like Cole started being born was that the drug causes major birth defects. Before thalidomide was banned worldwide in the 1960s more than 10,000 babies were born with serious deformities.
Thalidomide children were born all over the world, but the United States escaped the magnitude of the tragedy. Only 17 were ever born here because of a Food & Drug Administration doctor named Frances Kelsey. She didn't like some of the neurological research she saw on the drug back in 1960 and refused to let it on the market. Kelsey was later hailed as a hero and honored by then President John F. Kennedy.
PRESIDENT JOHN F. KENNEDY: I know that we're all most indebted to Dr. Kelsey.
BETTY ANN BOWSER: But now, 35 years later, the Food & Drug Administration has given tentative approval for thalidomide to be used to treat a disease that is extremely rare in the United States--leprosy. In September the FDA held a hearing where its advisory panel voted eight to two to allow thalidomide on the market for a first time. Cole and other thalidomide survivors say they are devastated, but they understand why supporters want it approved for marketing.
GISELLE COLE: Very scary, isn't it? It's really--it's a hard place to be in. It really is. It places us in a real moral dilemma. We don't want to see it. Yet, data would indicate that there are some benefits to it.
BETTY ANN BOWSER: The data she's talking about shows that thalidomide helps leprosy patients like these, who often lose fingers and toes as the disease literally eats away their body. In clinical trials the drug healed skins lesions and prevented new ones from forming. But thalidomide has also been shown effective in treating two AIDS-related illnesses, and because it seems to have an effect on the body's immune system, thalidomide may also be useful in treating certain forms of cancer and a wide array of autoimmune diseases, such as lupus and rheumatoid arthritis. These are diseases for which current drug therapy is limited. Dr. Patrick Hazelett is one of the doctors doing research on thalidomide at Rockefeller University in New York.
DR. PATRICK HAZELETT, Rockefeller University: What fascinates me and what fascinates doctors in general so much about this drug is that it appears to do dramatic things in some fairly specific disease states where other drugs fail. So I think there's an enormous amount of potential in this drug simply because of that phenomenon.
BETTY ANN BOWSER: If the FDA gives its final go-ahead, Celgene, a small drug company in New Jersey, will become the first in the United States allowed to market the drug. Even if thalidomide is eventually approved for the treatment of AIDS and other diseases CEO John Jackson sees it as having limited use.
JOHN JACKSON, CEO, Celgene: I think it's very important to understand, recognize that this drug should be reserved for very severely ill patients and people, in addition, whose lives are otherwise threatened. We don't wish to see this drug extensively used where other drugs are available. So I think the drug will be used to a great degree where it is the drug of last resort.
BETTY ANN BOWSER: But the American Academy of Pediatrics and even some doctors on the FDA's own advisory committee are concerned thalidomide will not just be a drug of last resort. Dr. Joel Mindel, a pharmacologist at Mt. Sinai Hospital in New York, voted for approval with great apprehension.
DR. JOEL MINDEL, Pharmacologist: There's no question that the more the drug is used the more chance there is for error. And patients might get the drug for perfectly valid uses and might give it to a friend who needs it.
BETTY ANN BOWSER: Randy Warren is chief executive officer of Thalidomide Victims of Canada, which has over 100 members. He also testified at the FDA hearing.
RANDY WARREN, Thalidomide Victims of Canada: There's going to be thalidomide babies born in the United States and in Canada. Once the United States has legalized this, as soon as that happens, then more tablets will be available. And doctors, given so much discretion from off-label use, frightens me to no end.
BETTY ANN BOWSER: Warren is not alone in his concern about so-called "off-label" use, the practice of prescribing drugs for treatments not approved by the FDA. Dr. Lewis B. Holmes, who also attended the FDA hearing, is an expert on birth defects who teaches at the Harvard Medical School.
DR. LEWIS B. HOLMES, Harvard Medical School: The way things are set up now there's not going to be a restriction that says the physician couldn't turn around and prescribe it for whatever your other good idea was as to what else it might benefit. And it becomes available for other reasons, even though the publicity and the application submitted by the manufacturer will talk only about leprosy. But it'll get it into the marketplace, and then if there's no restriction on who can prescribe, anybody could write a prescription for whatever.
BETTY ANN BOWSER: But Celgene CEO Jackson says his company and the FDA will make thalidomide the most regulated drug in the history of the United States. The company plans to require drugstores to register to buy thalidomide. Pharmacists would not be allowed to dispense more than a month's supply at a time. Automatic refills would not be allowed, and all patients would have to sign a national registry. Celgene CEO Jackson says he would like to put a picture of a thalidomide baby on the packaging and require special restrictions for women.
JOHN JACKSON: Needless to say, they need to have a pregnancy test to ensure they're not pregnant. And I'm sure the FDA is going to encourage various contraceptive methods and also an informed consent--not only an audio but also a video informed consent.
DR. LEWIS B. HOLMES: When I heard at the FDA hearings people saying, oh, well, we'll have an informed consent document, and she can read and sign. Well, there is a small group of people who will be influenced by informed consent documents, but most people aren't. They don't read them, or if they read them, they don't understand them. That's not good enough.
BETTY ANN BOWSER: What Holmes and other critics want the FDA to do is regulate who can prescribe thalidomide. That would mean only a certain specific number of physicians could dispense it. The NewsHour asked FDA officials for an on-camera interview, but the agency declined our request, saying it preferred not to comment until a final decision is made. But in its tentative approval letter to Celgene the FDA said, "The issue now is: How can you manage the down sides of this drug? That is the challenge." Celgene says the ultimate answer to that question will come when the company is able to make an entirely new thalidomide derivative drug that does not cause birth defects. The company has already made more than 100 new compounds or analogs from the original thalidomide drug. Sol Barer is president of the company.
SOL BARER, President, Celgene: The new drugs will be targeted toward what's known as inflammatory conditions. This is: lupus; multiple sclerosis; non-insulin-dependent diabetes, for which there are approximately greater than 10 million patients in the United States; psoriasis; many of the complications associated with AIDS.
BETTY ANN BOWSER: Until technology finds a new thalidomide drug that does not cause unacceptable side effects, thalidomide survivors like Giselle Cole are worried what will happen.
GISELLE COLE: I'm saying be very careful, be very careful. And, please, don't be cocky because quite often we hear, well, we appreciate and we understand the suffering that the past has caused but we think it can do da-da-ta-da-ta-da--this is something that didn't need to happen. This wasn't an accident of God. This was an accident by man. And I'm not a parent; I can't begin to describe what my parents must have felt, particularly my mother. I can only imagine. We certainly never want to have someone else experience that.
BETTY ANN BOWSER: Celgene says it expects final FDA approval within the next year to market the original thalidomide drug for the treatment of leprosy, and both the FDA and the company are on record promising to do everything they can to prevent the tragedy of 35 years ago from happening again.