The targeted agent, Zelboraf® (vemurafenib), which is approved for the treatment of melanoma, provided high anti-cancer activity among patients with hairy-cell leukemia that had stopped responding to prior therapies. These results were recently published in the New England Journal of Medicine.

Hairy-cell leukemia is a type of cancer in which too many abnormal immune cells are produced. It is considered a slow-growing type of leukemia that occurs more commonly in older men. The cancer cells have a “hairy” appearance under the microscope.

A genetic mutation referred to as the BRAF V600E mutation is thought to be an involved the excessive replication of hairy cell leukemia, as it is a common mutation in this type of cancer. Zelboraf binds to a specific site in cells that blocks the excessive replication caused by the BRAF V600E mutation.

Researchers from Italy and the United States conducted clinical trials to evaluate Zelboraf among patients with hairy cell leukemia that had stopped responding to prior standard therapy. Patients received 16-18 weeks of therapy in the form of an oral pill, twice daily.

Overall anti-cancer responses were achieved in 96% of patients in the U.S after a median of 12 weeks, and 100% of patients in Italy after a median of 8 weeks. At one year, 91% of patients were alive, while 73% of patients were alive with no progression of cancer. Also, side effects were reported as mainly mild.

The researchers concluded that “a short oral course of Zelboraf was highly effective in patients with relapsed or refractory hairy-cell leukemia.”

Reference: Tiacci E, Park J, De Carolis L, et al. Targeting mutant BRAF in relapsed or refractory hairy-cell leukemia. New England Journal of Medicine. Online September 9, 2015. 2015DOI: 10.1056/NEJMoa1506583

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