New Heart Failure Gene Therapy Unveiled

Gene therapy stabilizes cardiac function for heart failure patients

/ Author:  / Reviewed by: Joseph V. Madia, MD

(RxWiki News) About 5.8 million Americans suffer from heart failure with 670,000 new cases diagnosed each year. About 20 percent of those diagnosed die within a year. A new treatment might help better those odds.

Researchers at Mount Sinai School of Medicine have discovered a gene therapy that can improve cardiac function in those with heart failure.

In the second phase of the trial, patients received a high dose of a therapy called SERCA2a, that has significantly reduced cardiovascular hospitalizations, addressing a critical unmet need. SERCA2a is delivered through an inactive virus that acts as a medication transporter into cardiac cells.

"Discuss options with your physician if you suffer from heart failure."

This 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 received the SERCA2a demonstrated improvement or stabilization.

Dr. Roger J. Hajjar, M.D., 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 for the Mount Sinai School of Medicine, said few treatments had improved outcomes for heart failure patients in the last decade, and that the new treatment could become a novel target.

The CUPID (Calcium Up-regulation by Percutaneous administration of gene therapy In cardiac Disease) trial was a randomized, double-blind, placebo-controlled study, which enrolled 39 patients with advanced heart failure to study the safety and efficacy of SERCA2a. Patients were randomly received SERCA2a gene delivery in one of three doses or a placebo, and results were evaluated over the next year. The treatment is delivered directly to the patient's heart during a outpatient cardiac catheterization procedure.

Those in the high-dose SERCA2a group demonstrated improvement and stabilization in symptoms and overall heart function. There was also a dramatic reduction in cardiovascular hospitalizations, averaging 0.4 days versus 4.5 days in the placebo group.

The trial was funded by Celladon Corporation, which was co-founded by Dr. Hajjar. Dr. Hajjar has an equity interest in Celladon Corporation and participates on its Advisory Board. The research was published in American Heart Association journal Circulation.

Reviewed by: 
Review Date: 
June 30, 2011
Last Updated:
July 2, 2011