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Thalidomide: Controversial Treatment for Multiple Myeloma

March 10, 2006

The once-banned drug thalidomide appears to improve treatment for patients battling the blood cancer multiple myeloma, a new study shows, but the jury is still out on its impact on their long-term survival. Thalidomide was taken off the market in the 1960s because women who took it during pregnancy had a much higher rate of severe birth defects. However, studies suggesting it might help against cancer led to its reintroduction -- with strict controls -- in 1998.

An Italian team of researchers at the University of Turin added thalidomide to standard therapy (melphalan plus prednisone) for newly diagnosed cases of the blood cancer multiple myeloma in older patients.

As reported in the March 11 issue of The Lancet, there was a 76 percent treatment response rate for the 129 patients who got thalidomide compared to a 47.6 percent response rate for the 126 participants who did not get the drug. Patients who received thalidomide in their treatment also boosted their chance of event-free survival over two years -- 54 percent for those who got thalidomide vs. 27 percent for those who did not.

However, that finding is at odds with the result of an American trial reported in Thursday's New England Journal of Medicine. While both studies found that thalidomide provided short-term therapeutic benefit, the University of Arkansas study found that patients taking the drug actually relapsed sooner than patients not on the therapy, eliminating any benefit in terms of added survival.

The Italian researchers agree that more study is needed to sort this out.

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