New drug, Vemurafenib, doubles survival of metastatic melanoma patients

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 melanoma 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 Medicine, 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 metastatic melanoma harboring a BRAF mutation."