Mount Sinai Researchers to Treat Advanced Heart Failure Through Gene Therapy

      According to the U.S. Centers for Disease Control and Prevention, about 5.8 million Americans suffer from heart failure, with over half a million new cases diagnosed each year. Fatality of this disease is one in five within the first year of diagnosis. Often treated with aggressive medical and device therapy, heart disease has no cure. Symptoms include shortness of breath, exhaustion, and extremity swelling in the ankles, feet, legs, and occasionally the abdomen.

       The Mount Sinai Medical Center, which encompasses both the Mount Sinai Hospital and Mount Sinai School of Medicine, is one of the few dual-institutions nestled within a hospital in the nation. Established in 1968, the Mount Sinai Medical Center ranks among the top 20 medical schools both in NIH funding and according to the US News & World Report.

       Recently researchers at Mount Sinai School of Medicine have developed a gene therapy that stabilizes and improves cardiac function in severe heart failure patients. This therapy, called SERCA2a, is delivered through an inactive medication-transporting virus into the cardiac cells, which results in production of an enzyme within the cells that encourages heightened pump efficiency.

        The phase II trial that took place involved 39 patients with severe heart failure, and was enacted to study the safety and efficacy of SERCA2a. After one year, this CUPID (Calcium Up-regulation by Percutaneous administration of gene therapy In cardiac Disease) trial demonstrated improvement or stabilization in high-dose patients.

     Gene therapy with SERCA2a was also found to be safe with zero increase in adverse events relative to placebo.

     Patients in the high-dose SERCA2a group showed overall improvement/stabilization in heart function, biomarker activity, and ventricular mechanics.

       "Few treatment options have shown such improved clinical outcomes in this patient population in the last decade," says Roger J. Hajjar, MD, Research Director of Mount Sinai's Weiner 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"

(image of Roger J. Hajjar, MD courtesy of The Mount Sinai Medical Center)

        He also commented that through the success of the use of adeno-associated virus vectors ( inactive medication transport virus), "this study ushers in a new era for gene therapy for the treatment of failing hearts."

        The vast potential of the cardiac-specific target was discovered by the Mount Sinai team led by Dr. Hajjar in 1999. Since then, exploration and development has been pursued as a treatment-delivered gene therapy for cardiovascular disease. The CUPID trial, co-founded by Dr. Hajjar, is funded by Celladon Corporation.

Article Courtesy of The Mount Sinai Medical Center

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