The Case for Thalidomide

NEARLY 30 YEARS AFTER THE DRUG THALIDOMIDE WAS yanked from the consumer market, the mere mention of it can still evoke shudders of horror from those who came to know of its side effects. Marketed as a tranquilizer, thalidomide turned out to be one of the most potent causes of birth defects ever found. Babies born to women who took it during early pregnancy were born with terrible deformities, including missing or seriously misshapen limbs.

But now the long-feared drug has also become a source of hope. Doctors have known for years that thalidomide is among the most effective treatments for leprosy. And last week a research team from Johns Hopkins reported in the New England Journal of Medicine that the drug can also improve the survival rate of patients who get bone-marrow transplants, which are used to treat potentially fatal disorders including aplastic anemia and some blood cancers.

The most common complication of such transplants, though, is graft-vs.-host disease (GVHD), a potentially fatal reaction in which the foreign tissue tries to reject its new body (rather than the other way around). Researchers have found that thalidomide seems to keep that reaction in check. Compared with other treatments for GVHD, the drug is relatively benign — as long as the patient is absolutely certain she’s not pregnant.