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A New Gene Therapy Shows Promise For Treating Severe Heart Failure

June 7, 2010

According to the U.S. Centers for Disease Control & Prevention, about 5.8 million Americans suffer from heart failure. About 670,000 new cases are diagnosed each year, and 1 in 5 people die within one year of diagnosis. Heart failure is most often treated with aggressive medical and device therapy, but has no cure. Researchers at Mount Sinai School of Medicine in New York have developed a new gene therapy to reverse advanced heart failure—SERCA2a (produced as MYDICAR®) is designed to stimulate production of an enzyme that enables the failing heart to pump more effectively. In a Phase II study, SERCA2a injection through a routine, minimally invasive cardiac catheterization, showed clinical benefit in treating this patient population and decreasing the severity of heart failure. The data were presented at the Heart Failure Congress of the European Society of Cardiology in Berlin. "SERCA2a met the primary endpoints and appears to be safe and effective in people with advanced heart failure," said trial investigator Jill Kalman, MD, Associate Professor, Medicine, Cardiology, Director of the Cardiomyopathy Program, Mount Sinai School of Medicine. "There is a significant unmet need for treatments in this patient population, and these data indicate that SERCA2a is a promising option for them." Patients in the SERCA2a group of the randomized, double-blind, placebo-controlled study, demonstrated improvement or stabilization in symptoms, heart function and severity of heart failure. They also saw an increase in time between cardiovascular events and a decrease in frequency of events. SERCA2a was found to be safe, with no increases in adverse events, disease-related events, laboratory abnormalities or arrhythmias compared to participants who received a placebo instead.

SERCA2a was developed by a team led by Roger J. Hajjar, MD, Research Director of Mount Sinai's Wiener Family Cardiovascular Research Laboratories and the Arthur & Janet Ross Professor of Cardiology, Medicine, and Gene and Cell Medicine, Mount Sinai School of Medicine. The team discovered the landmark potential of the treatment in 1999 and has been pursuing its potential as a gene therapy target in state-of-the-art specially built pre-clinical laboratories at Mount Sinai. "Mount Sinai Heart is committed to developing ground-breaking therapies and bringing them from bench to bedside," said Valentin Fuster, MD, Director of Mount Sinai Heart, the Zena and Michael A. Wiener Cardiovascular Institute and the Marie-Josee and Henry R. Kravis Center for Cardiovascular Health, The Mount Sinai Medical Center. "We look forward to further study of this important treatment."