ELIZABETH FARNSWORTH: Next, the return of thalidomide. We begin with an updated version of a Betty Ann Bowser report we aired last fall.
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 has shortened arms and only three fingers on each hand because 37 years ago her mother took a drug called thalidomide while she was pregnant with Giselle. At the time, thalidomide was widely prescribed for morning sickness. 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. Most of the children were born in Europe, where thalidomide was readily available, but there were also hundreds of others born with birth defects in Canada and in the Caribbean. The only country to escape the tragedy was the United States. Only 17 thalidomide babies were ever born here because an FDA named Frances Kelsey didn't like some of the neurological research she saw on the drug back in 1960, and she refused to let it on the market. But now, 40 years later, the FDA has given final approval for its use to treat an almost unheard of disease in this country --leprosy. Cole and other thalidomide survivors are concerned.
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: In clinical trials thalidomide has been shown effective in treating not just leprosy but two AIDS-related illnesses, and because it seems to have an effect on the body's immune system, thalidomide may also be effective in treating certain forms of cancer and a wide array of autoimmune diseases like 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: Celgene, a small drug company in New Jersey, is 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 besides leprosy, 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 has expressed concern that thalidomide will not just be a drug of last resort. And Randy Warren, CEO of the organization Thalidomide Victims of Canada, told the NewsHour last year he's worried about dire consequences.
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: But Celgene CEO Jackson says his company and the FDA will make thalidomide the most regulated drug in the history of the United States. And the company says the ultimate answer is to find a new thalidomide compound drug, one that does not produce birth defects. 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: Dr. Stephen Oliver is working with one of those new compounds at Rockefeller University. One group of white laboratory rats was injected with chemicals that cause rheumatoid arthritis. Eight days later their joints are swollen and sore. The rates can barely move their legs. Another group of rats given the same chemical was also given the new thalidomide compound, or analog, every day.
DR. STEPHEN OLIVER: As you can see, these rats injected at the same time as their sister mates over there with the stimulus for arthritis have failed to show much in terms of arthritis. They're as frisky as the ones that received no injection for arthritis. There's some mild inflammation, but it's very minimal at best. They were given the analog on a daily basis, just like a human patient might take medicine on a daily basis.
BETTY ANN BOWSER: Now, like all the new compounds, the drug needs to be tested in human trials to see if it works without side effects. Researchers say a new thalidomide drug is at least five to ten years away. In the meantime, thalidomide survivor Cole is worried what will happen.
GISELLE COLE: I'm saying be very careful, be very careful. This is something that didn't need to happen. This isn'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: Cole is working with Celgene to develop informational videos about thalidomide for the public. Meanwhile, the drug will become available this fall.
ELIZABETH FARNSWORTH: And now to Phil Ponce, who talked last week with the Acting Commissioner of the FDA, Dr. Michael Friedman.
PHIL PONCE: Dr. Friedman, welcome.
DR. MICHAEL FRIEDMAN, Acting Commissioner, FDA: Thank you.
PHIL PONCE: Expand on why the FDA has approved a drug that is so dangerous.
DR. MICHAEL FRIEDMAN: For patients with a very rare complication of leprosy, patients who desperately need drug treatment that's effective, it's been recognized for some 30 years that thalidomide provides them measurable, meaningful benefits.
PHIL PONCE: You're approving it for leprosy and yet, once it's legally out on the market doctors can prescribe it for other things-treatment of symptoms pertaining to AIDS and possibly cancer and other things that were alluded to in the piece. So it could conceivably go well beyond people who suffer from leprosy, yes?
DR. MICHAEL FRIEDMAN: That's correct. Physicians can prescribe it, but there have been safeguards built in to try and deal with the very serious concerns that were documented in the piece that you just presented.
PHIL PONCE: And tell us about some of those safeguards.
DR. MICHAEL FRIEDMAN: Well, I think it is fair to say that this is in a way the most regulated product that we've ever had. There's a very intensive, a very ambitious program that consists of first of all pregnancy testing for women of child-bearing age, very intensive birth control practices, not just for women who take this medicine but also for men, a formal educational program for physicians, pharmacists and health care workers who are prescribing and dispensing the medicine, and then a very effective educational program we think for patients who will be taking it.
As a matter of fact, Ms. Cole, the individual who was featured in your piece, speaks very powerfully and very eloquently on the videotape that's given to every patient to view before they take this. There's then registration not just for the patients but the physicians and pharmacists, and finally there's a very formal and very extensive informed consent procedure, so that the system is multi-layered. It's ambitious, but it aims to assure that these pills will be properly taken.
PHIL PONCE: Going back to the requirements and the patients, the birth control requirements, you're saying what, that female patients have to be on birth control, and, likewise, male patients have to, what, use condoms?
DR. MICHAEL FRIEDMAN: That's correct. As a matter of fact, female patients must agree to actually use two forms of birth control simultaneously, one that we consider very effective like birth control pills or implantable birth control devices, intrauterine devices, or if they've had a hysterectomy, that would obviously qualify. But in addition to that they must use a barrier method, such as a diaphragm or cervical cap. For men, they just use condoms with every sexual encounter.
PHIL PONCE: And how does a doctor know his or her patients are following those procedures?
DR. MICHAEL FRIEDMAN: Well, the educational activities for the patients are really very intensive, and I think patients understand what's at stake. In addition, there's mandatory pregnancy testing for the women who take the medication either once every two weeks or once every four weeks. Unless the woman has a negative pregnancy test, that prescription cannot be filled, nor can it be renewed in the future.
PHIL PONCE: So there are requirements on doctors, patients. How do you know that-how can you be assured that everyone up and down the line is going to follow all those rules?
DR. MICHAEL FRIEDMAN: Well, I think the materials are so effective in conveying the risks, and that the risks are so clear and so important that that would be one very important way, but, in addition, the company has given us very extensive assurances about how they will be following this, but, in addition, the Food & Drug Administration will be carefully monitoring how many patients, for what diseases, under what circumstances we're going to be watching this very, very closely.
PHIL PONCE: One of the points that was made in the introductory piece from one of the victims in Canada was in spite of your best efforts, somewhere along the line there's going to be a pregnancy, and somebody is going to be born with birth defects. Is that a reasonable fear?
DR. MICHAEL FRIEDMAN: It is a fear. I do not accept that this will happen. I do understand and I do respect those who say that there is a likelihood, there's a chance of it. I know there's a chance of it. We're going to do everything we can to have a zero incidence of malformed children.
That's going to be very difficult, and I don't underestimate how difficult it is, but our commitment and our pledge to the children who might possibly be harmed is so great that we're going to do everything that we can if we find there's even a single episode where these built-in safeguards have not been effective. We're going to re-evaluate the entire program to find out what might have failed in our systems, as they currently exist, and see what we can do to improve them. So we're really committed not in a haphazard way, not in an idealistic way, but in a practical way, to making sure that the children that we care about are best protected.
PHIL PONCE: Will that re-evaluation possibly include pulling it off the market if somehow the thing were not working?
DR. MICHAEL FRIEDMAN: I think that all options are available to us. Certainly the Food & Drug Administration has demonstrated an ability to remove products from the market when guidelines can't be followed. I don't envision that would happen here. I do not. But we have all options open and available to us.
PHIL PONCE: One of the things that was pointed out at the end of the piece was the possibility that new drugs are being developed that would accomplish what thalidomide-the positive things that thalidomide can accomplish without some of those side effects. How likely is that?
DR. MICHAEL FRIEDMAN: Well, I don't know how likely is it to have something that will be as effective and as successful, but the research is terribly important, and we are interested in watching the outcome of that. I do think, though, it's fair to say that no medication that has benefits is completely free of side effects.
And while it may be possible to remove the damage to developing children, there may be other side effects that are encountered that could be equally or even more severe. Therefore, I don't think it's realistic to hope that you'll have an effective treatment that's completely free of side effects. But we have to keep striving to minimize those side effects.
PHIL PONCE: We've touched on this, but in spite of everything that the FDA is doing, are you still a little bit nervous about putting this back on the market?
DR. MICHAEL FRIEDMAN: I think that putting this product back on the market at this time requires a real recognition of what's at stake. These are desperately ill patients. These are patients in real need, but we have an enormous commitment to not have this side effect for children so that we view this with real care, with real intense interest, and we're going to be following it very closely.
PHIL PONCE: Dr. Friedman, thank you for being with us.