New Uses For Thalidomide Yielding Valuable Lessons

Sidebar: Researchers Explore Thalidomide's Therapeutic Potential Firms are focusing on getting the teratogen to market to treat serious diseases; if successful, it may inspire fresh looks at other compounds. Thirty-five years after the effects of thalidomide horrified the world, the drug is teaching researchers a whole new set of lessons. This time, though, the message is positive: Working together, companies and the Food and Drug Administration (FDA) can indeed pursue important new compounds

Written byKathryn Brown
| 8 min read

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Sidebar: Researchers Explore Thalidomide's Therapeutic Potential

Sold outside the United States as a sedative and morning sickness treatment in the 1950s and 1960s, thalidomide caused thousands of babies to be born with malformed arms and legs. In the wake of the disaster, FDA created guidelines for preclinical animal tests to learn if a new compound causes birth defects before it ever enters humans. Today, most companies abandon would-be pharmaceuticals at the first sign of birth defects in animal models.

But that could change-at least for drugs that treat serious disease. Ironically, thalidomide is now helping to open research opportunities for teratogenic (birth-defect-causing) drugs that fill a true market need. Several companies are pursuing thalidomide because it appears to moderate immune system reactions that characterize diseases like cancer and AIDS (see story on page 8).

MARKET NICHE: Celgene president Sol Barer says "the balance is in favor of" having thalidomide available ...

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