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:


Penn Medicine is one of the world's leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of the Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania (founded in 1765 as the nation's first medical school) and the University of Pennsylvania Health System, which together form a $5.3 billion enterprise.

The Perelman School of Medicine has been ranked among the top five medical schools in the United States for the past 18 years, according to U.S. News & World Report's survey of research-oriented medical schools. The School is consistently among the nation's top recipients of funding from the National Institutes of Health, with $373 million awarded in the 2015 fiscal year.

The University of Pennsylvania Health System's patient care facilities include: The Hospital of the University of Pennsylvania and Penn Presbyterian Medical Center -- which are recognized as one of the nation's top "Honor Roll" hospitals by U.S. News & World Report -- Chester County Hospital; Lancaster General Health; Penn Wissahickon Hospice; and Pennsylvania Hospital -- the nation's first hospital, founded in 1751. Additional affiliated inpatient care facilities and services throughout the Philadelphia region include Chestnut Hill Hospital and Good Shepherd Penn Partners, a partnership between Good Shepherd Rehabilitation Network and Penn Medicine.

Penn Medicine is committed to improving lives and health through a variety of community-based programs and activities. In fiscal year 2015, Penn Medicine provided $253.3 million to benefit our community.