Researchers at Mount Sinai School of Medicine have found in a Phase II trial that a gene therapy developed at Mount Sinai stabilized or improved cardiac function in people with severe heart failure. Patients receiving a high dose of the therapy, called SERCA2a, experienced substantial clinical benefit and significantly reduced cardiovascular hospitalizations, addressing a critical unmet need in this population. The data are published online in the June 27 issue of the American Heart Association journal Circulation.
SERCA2a is delivered via an adeno-associated virus vectoran inactive virus that acts as a medication transporterinto cardiac cells. The therapy stimulates production of an enzyme within these cells that enables the heart to pump more effectively in people with advanced heart failure. After one year, patients who were administered a high dose SERCA2a demonstrated improvement or stabilization. Gene therapy with SERCA2a was also found to be safe in this sick patient population, with no increases in adverse events, disease-related events, laboratory abnormalities, or arrhythmias compared to placebo.
"Few treatment options have shown such improved clinical outcomes in this patient population in the last decade," said Roger J. Hajjar, MD, Research Director of Mount Sinai's Wiener Family Cardiovascular Research Laboratories, and the Arthur and Janet C. Ross Professor of Medicine, and Gene and Cell Medicine, Mount Sinai School of Medicine. "This study establishes a new paradigm for the treatment of heart failure by clinically validating SERCA2a as a novel target. In addition, by showing that adeno-associated vectors are safe to use in patients with advanced heart failure, this study ushers a new era for gene therapy for the treatment of failing hearts."
The CUPID (Calcium Up-regulation by Percutaneous administration of gene therapy In cardiac Disease) trial is a randomized, double-blind, placebo-controlled study, which enrol
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