New drug, Vemurafenib, doubles survival of metastatic melanoma patients

March 1, 2012, University of Colorado Denver

A report published this week in the New England Journal of Medicine shows that the 50 percent of metastatic melanoma patients with a specific genetic mutation benefit from the drug Vemurafenib – increasing median survival from about 6 months to 15.9 months. In patients who responded, the drug stopped cancer progression for a median 6.7 months.

"For patients with a BRAF V600 mutation, this drug is a breakthrough. Not a cure, but a major breakthrough," says Karl Lewis, MD, investigator at the University of Colorado Cancer Center, associate professor at the University of Colorado School of , and one of the study's authors.

Lewis notes that until about 18 months ago, no drug existed for metastatic melanoma — the most dangerous form of skin cancer — that was proven to extend survival past that of patients who chose not to treat the disease. The CU Cancer Center is a leading treatment center for metastatic melanoma, and has been instrumental in enrolling patients in trials of this new category of melanoma drugs — BRAF inhibitors.

The BRAF mutation is a known oncogene – a gene that when mutated causes cancer. Specifically, the BRAF V600 mutation signals a cell to grow without bounds. Vemurafenib is a BRAF inhibitor. The mutation turns cancer on and Vemurafenib turns it off.

And turning off BRAF in the approximately 100,000 patients diagnosed worldwide each year with BRAF-positive metastatic melanoma more than doubles their time of survival.

"Rarely do we see results this dramatic," says Lewis. "This represents a new standard of care for patients with harboring a BRAF mutation."

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