Skip to Content

‘Bright Day for Many Melanoma Patients’

Approval of BRAF Inhibitor Another Step in Personalized Cancer Medicine Journey

August 18, 2011 | Cynthia Floyd Manley

News that the Food and Drug Administration had approved a new targeted drug for advanced melanoma was heralded as good news this week by physician-scientists at Vanderbilt-Ingram Cancer Center, which was involved in many of the studies that helped lead to the approval.

“This is a bright day for many melanoma patients,” said Dr. Jeffrey A. Sosman, professor of Medicine and leader of Vanderbilt-Ingram’s melanoma program. “But it is only the start, and we have to continue to enroll patients onto clinical trials in order to build upon this exciting advance.”

The drug vemurafenib (previously PLX4032) has generated a lot of excitement because it targets a specific genetic mutation found in some 40-50% of patients with metastatic melanoma. The decision to approve the drug came on Aug. 17.

Vemurafenib (marketed as Zelboraf)  is not a cure — not all patients with this genetic change, known as BRAF V600E, respond to the drug, and many patients who do eventually see their disease recur.

But the drug has made a difference for many patients in both length and quality of life, said Dr. Igor Puzanov, assistant professor of Medicine and a co-investigator in the vemurafenib trials.

“I’ve seen patients stop having pain, able to go back to work and do the things they enjoy, and the drug does prolong life,” Puzanov said. He noted that while median survival (the point at which half the patients on a study are still alive) was nine months with vemurafib, some patients who were involved in the earliest of human studies are still being followed two and three years later.

The next step in clinical research will be to develop combinations of therapy that can increase effectiveness, decrease side effects and prevent or at least delay the amount of time for the tumor to develop resistance, he said.

Last year, Vanderbilt-Ingram became one of the first cancer centers to routinely test all melanoma patients for genetic mutations that could make a difference in treatment approach, including BRAF mutations in patients with melanoma. It is part of its Personalized Cancer Medicine Initiative, which also includes lung and breast cancers. As a leader in the development of personalized cancer medicine, Vanderbilt-Ingram also has launched My Cancer Genome, a free online tool for researchers and doctors that links the latest knowledge about cancer genetics with information about how specific DNA mutations can impact treatment decisions, along with links to clinical trials at Vanderbilt-Ingram and around the world.

Additional information:

In this video, Sosman and other leaders at Vanderbilt-Ingram discuss the promise of this “personalized” approach to treating cancer patients: