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Second chance for thalidomide

Infamous for causing birth defects in the late 1950s, thalidomide is making a comeback as a drug to help patients with multiple myeloma, cancer of the plasma cells in the bone marrow.

Thalidomide, a sedative and former morning-sickness remedy, also turns out to be an effective antiangiogenetic—that is, it blocks the formation of new blood vessels. This probably explains why so many “thalidomide babies” were born with shortened arms or legs and, ultimately, why the drug was banned worldwide in 1962.

Keeping tumors at bay

After that debacle, thalidomide was all but forgotten except by a few researchers. They eventually found that the drug could boost the immune system and interrupt blood flow, thereby helping to starve cancer tumors by denying them oxygen and nutrition.

In multiple myeloma, an incurable and usually fatal disease, tumors develop in the bone marrow. As a result, the body no longer produces enough blood cells. This in turn causes anemia, excessive blood loss from minor cuts and low resistance to infection. Myeloma also stimulates bone-dissolving cells and can cause fractures.

Until recently, standard treatment for multiple myeloma has been comprised of chemotherapy, radiation, transfusions and bone marrow transplants—often with poor long-term results. But when thalidomide was given in small-scale experimental trials at the University of Arkansas in the late 1990s, 60 percent of end-stage (that is, near death) patients were still alive two years later, and 20 percent were disease-free.

A second small study at the Mayo Clinic, released in 2000, confirmed the Arkansas findings. Thalidomide, in combination with dexamethasone, has become the usual first treatment for many patients, especially those who will be receiving another innovative treatment, stem cell transplantation.

Strong warnings

Researchers are not sure how thalidomide works against myeloma. And despite encouraging results, the drug is considered so dangerous that the U.S. Food and Drug Administration has released tough new guidelines on its use—by men as well as by women.

Women should:

  • not take thalidomide if pregnant or nursing
  • have a doctor give them a pregnancy test before treatment starts, then monthly during treatment and four weeks after treatment ends
  • abstain from sexual intercourse from a month before treatment starts until a month after treatment ends, unless she has had a hysterectomy

Men should:

  • abstain from sexual intercourse from a month before treatment starts until a month after treatment ends

Both should:

  • never give thalidomide to anyone else
  • follow dosage instructions to the letter
  • be aware that thalidomide causes drowsiness and may cause nerve damage