Use of an experimental targeted drug to treat metastatic melanoma tumors with a specific genetic signature was successful in more than 80 percent of patients in a phase 1 clinical trial. Results of the trial of PLX4032, an inhibitor of a protein called BRAF that is overactive in more than half of all melanomas, appear in the August 26 New England Journal of Medicine. The role in melanoma of the BRAF mutation, which keeps the protein constantly activated and driving cell growth, was discovered in 2002 by researchers at the Sanger Institute in Britain.
The new study was led by Keith Flaherty, MD, formerly at the University of Pennsylvania Abramson Cancer Center, now at Massachusetts General Hospital, and Peter O’Dwyer, MD, professor of Medicine, began to explore whether drugs targeting the mutation might interfere with tumor growth. The Abramson Cancer Center will be enrolling patients in a larger trial involving BRAF inhibitors, which will be led by Lynn Schuchter, MD, professor of Medicine and Ravi Amaravadi, MD, assistant professor of Medicine.
For more information about the NEJM study, see the Mass General press release:
For more information about the study and ongoing related studies at Penn, see the Abramson Cancer Center website:
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