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Taking thalidomide out of rehab

Nature Medicine volume 16, pages 370–372 (2010)Cite this article

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Thalidomide, a drug reviled in the 1960s for its teratogenic effects, has been revived in recent years for cancer and leprosy therapy. A study now finds another use for this drug in vascular disease, providing further insights into the drug's mechanisms of action (pages 420–428).

The thalidomide tragedy of 1957–1961 was one of the greatest medical disasters of modern times. Worldwide the drug was prescribed to pregnant women to alleviate morning sickness, but it resulted in widespread spontaneous abortion and severe birth defects in over 10,000 babies, 40% of whom died before one year of age1. Many thalidomide victims are still alive, and their characteristic limb deformities are a potent reminder to society of the cost of an incomplete understanding of drug mechanisms and metabolism and inadequate in vivo testing before clinical use. Thalidomide's safety was originally tested on mice, but, when administered to pregnant mice, the drug does not reach teratogenic levels in embryos as it does in other vertebrates.

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Figure 1: Thalidomide bypasses the requirement of the TGF-β1–endoglin–Alk-1 signaling pathway for vascular stability.

Kim Caesar

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Authors and Affiliations

  1. Rosemary J. Akhurst is at the Helen Diller Family Comprehensive Cancer Center and Department of Anatomy, University of California–San Francisco, San Francisco, California, USA.,

    Rosemary J Akhurst

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  1. Rosemary J Akhurst
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Correspondence to Rosemary J Akhurst.

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Akhurst, R. Taking thalidomide out of rehab. Nat Med 16, 370–372 (2010). https://doi.org/10.1038/nm0410-370

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