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AROUND THE MEDICAL CENTER :: WINTER 2014
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Melanoma drug shows promise in shrinking tumors


By Dagny Stuart
January 2011

A new drug used to treat metastatic melanoma patients who have the genetic mutation known as BRAFV600E demonstrated significant tumor shrinkage in the majority of patients during a clinical trial.

Data from the Phase I trial of the drug PLX4032, developed by Plexxikon Inc. and Roche Pharmaceuticals, showed that nearly all patients with the mutation who were treated with the drug showed some response, and 81 percent of patients had tumor shrinkage of at least 30 percent. The data were published in the New England Journal of Medicine.

Melanoma, a form of skin cancer, is curable when caught in its early stages but it can be lethal when it metastasizes to other areas of the body. Fewer than 10 percent of patients with metastatic melanoma are still alive five years after diagnosis, according to the National Cancer Institute.

“In the more than 20 years that I have been taking care of melanoma patients, I have not seen this kind of patient response to a therapy,” said Jeffrey Sosman, M.D., director of the Melanoma Program at Vanderbilt-Ingram Cancer Center.

“We finally have a way to identify patients with a specific genetic mutation who are most likely to benefit from a treatment, and a drug that targets that mutation. This is the promise of personalized medicine and Vanderbilt's patients are benefiting from this approach to cancer treatment.”

The PLX4032 drug, which is administered as a pill, is currently in parallel and ongoing Phase II and Phase III trials. The drug is a small molecule that is selective for a key oncogenic driver in melanoma and other cancers. Sosman is leading the Phase II trial.

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