Yes, thalidomide. The word has been synonymous with horror since the late 1950s, when 10,000 deformed babies were born worldwide to mothers who had used the drug to alleviate morning sickness. The sight of those children–some lacking eyes or ears, many born with tiny flippers for limbs–still haunts drug regulators. Yet the FDA is now poised to let thalidomide onto the U.S. market–and thousands of very sick people are looking to it for relief.
Thalidomide’s rehabilitation started almost as soon as it was shelved in the early 1960s. In studying the drug, researchers discovered that it blocks the formation of new blood vessels, while slowing the immune system’s production of a troop-rallying chemical called tumor necrosis factor alpha (TNF-a). The first effect had disastrous consequences for the developing fetus–limbs can’t grow without blood vessels–but it had potential benefits for people with cancer and other conditions in which vessel growth causes trouble. Likewise, the drug’s ability to slow the production of TNF-a was a potential boon to people with diseases like type-1 diabetes, in which the immune system attacks the body’s own tissues.
By the 1970s, researchers had found that thalidomide could forestall the fever, joint pain and skin nodules associated with leprosy (presumably by modulating TNF-a). Further study has shown that it can ease the symptoms of lupus, multiple sclerosis and rheumatoid arthritis, and can combat AIDS-related wasting. Researchers are also studying it as a possible treatment for brain and prostate tumors, not to mention macular degeneration and diabetic retinopathy, the two leading causes of blindness in adults.
The FDA is approving thalidomide only as a treatment for leprosy complications. But once it reaches the market (probably early next year), doctors will be free to prescribe it at will. And as the drug comes into wider use, the odds increase that unborn babies will be exposed. Other drugs can damage fetuses, but thalidomide poses extreme risks, so Celgene Inc., the New Jersey-based firm that will market the drug, plans extraordinary safeguards. No prescription will be filled without a statement certifying that the doctor and patient have discussed the hazards. Prescriptions will provide only four weeks’ worth of medication (no automatic refills). Celgene will advise women to take a pregnancy test before starting the drug and to use two forms of birth control while on it. And for good measure, each capsule will carry a warning symbol (photo). The package insert may even show pictures of thalidomide babies.
If we’re lucky, those grim warnings will someday be unnecessary. Researchers are now working to develop thalidomide-like drugs that yield the same benefits without the hazards. Until then, we can only hope that triumphs like Vicki Walton’s don’t come at too high a cost.
