Novel Targeted Therapy for Melanoma Shows Promise

Majority of patients on therapy targeting mutated BRAF gene have tumor regression

WEDNESDAY, Aug. 25 (HealthDay News) -- Therapy targeting the V600E BRAF mutation found in many melanoma tumors can result in complete or partial tumor regression in most patients, according to a study in the Aug. 26 issue of the New England Journal of Medicine.

Keith T. Flaherty, M.D., of the University of Pennsylvania in Philadelphia, and colleagues tested a potential therapy -- PLX4032 -- targeting the V600E mutation in the gene encoding the serine-threonine protein kinase B-RAF (BRAF). Fifty-five patients (49 with melanoma) participated in a dose-escalation phase, and then 32 patients with metastatic melanoma were given 960 mg of PLX4032 twice daily in an extension phase.

In the dose-escalation group, among the 16 melanoma patients with the mutation who received 240 mg or more of PLX4032 twice daily, the researchers found that 10 had a partial response and one had a complete response. Of the 32 patients in the extension group, 24 had a partial response and two had a complete response. For all patients, the estimated median progression-free survival was more than seven months.

"In our study, PLX4032 induced responses in the vast majority of melanomas caused by BRAF mutations, which constitute 40 to 60 percent of all melanomas. We do not yet know whether treatment with PLX4032 will improve overall survival; an ongoing phase 3 trial is addressing that question," the authors write.

The study was supported by Plexxikon and Roche Pharmaceuticals. Study authors reported financial ties to these companies.

Full Text (subscription or payment may be required)
Editorial (subscription or payment may be required)

Physician's Briefing