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:
http://www.massgeneral.org/about/pressrelease.aspx?id=1274
For more information about the study and ongoing related studies at Penn, see the Abramson Cancer Center website:
http://penncancer.org/penn_news.cfm?ID=1206
Penn Medicine is one of the world’s leading academic medical centers, dedicated to the related missions of medical education, biomedical research, excellence in patient care, and community service. The organization consists of the University of Pennsylvania Health System (UPHS) and Penn’s Raymond and Ruth Perelman School of Medicine, founded in 1765 as the nation’s first medical school.
The Perelman School of Medicine is consistently among the nation's top recipients of funding from the National Institutes of Health, with $580 million awarded in the 2023 fiscal year. Home to a proud history of “firsts,” Penn Medicine teams have pioneered discoveries that have shaped modern medicine, including CAR T cell therapy for cancer and the Nobel Prize-winning mRNA technology used in COVID-19 vaccines.
The University of Pennsylvania Health System cares for patients in facilities and their homes stretching from the Susquehanna River in Pennsylvania to the New Jersey shore. UPHS facilities include the Hospital of the University of Pennsylvania, Penn Presbyterian Medical Center, Chester County Hospital, Doylestown Health, Lancaster General Health, Princeton Health, and Pennsylvania Hospital—the nation’s first hospital, chartered in 1751. Additional facilities and enterprises include Penn Medicine at Home, GSPP Rehabilitation, Lancaster Behavioral Health Hospital, and Princeton House Behavioral Health, among others.
Penn Medicine is an $11.9 billion enterprise powered by nearly 49,000 talented faculty and staff.