Crescent Discovery Series

A reading room for breakthroughs and the curious mind

The birth-defect drug that came back to fight cancer

Thalidomide was pulled from markets worldwide in the early 1960s after causing severe birth defects in thousands of children.

Crescent Bioresearch29 September, 8:43 AMSinghal et al., NEJM, 1999 · University of Arkansas for Medical Sciences2 min read

Decades later, researchers at the University of Arkansas tried it in patients with multiple myeloma who had run out of other options, reasoning from theories about how tumors build new blood vessels that the same drug blamed for malformed limbs might starve tumors of their blood supply.

Bart Barlogie's team gave oral thalidomide to 84 patients with myeloma that had relapsed after other treatments, including high-dose chemotherapy in most cases. Thirty-two percent responded, including two complete remissions, in a disease where these patients had been expected to have few options left.

The result launched thalidomide and its newer chemical relatives, lenalidomide and pomalidomide, into standard myeloma treatment, where they remain core drugs today, combined with newer antibodies and transplants. Thalidomide itself is now dispensed only under strict pregnancy-prevention programs, a condition of its return, given the history that took it off the market in the first place.

Crescent Discovery Series cover: The birth-defect drug that came back to fight cancer

A banned drug became a cancer backbone.

Source

  1. Singhal, S., Mehta, J., Desikan, R., Ayers, D., Roberson, P., Eddlemon, P., Munshi, N., Anaissie, E., Wilson, C., Dhodapkar, M., Zeddis, J., & Barlogie, B. (1999). Antitumor activity of thalidomide in refractory multiple myeloma. New England Journal of Medicine, 341(21), 1565–71. https://doi.org/10.1056/NEJM199911183412102
Crescent Discovery Series

Follow the series

New discoveries are published on our channels every day.

Keep going

Three more numbers