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Gene Therapy Shows Long-Term Benefit for Heart Failure

Heart monitor read out

(brykmantra/Flickr)

A therapy using genes to induce production of an enzyme for heart cells to restore their use of calcium was found to help heart failure patients in a clinical trial reduce their incidents of cardiac problems over a three-year period. Researchers at Mount Sinai Health System in New York, with colleagues from the biotechnology company Celladon Corp. in San Diego, University of California in San Diego, and University of Pennsylvania reported their results today at a meeting of American Heart Association in Dallas.

The paper discusses findings from a clinical trial of a candidate gene therapy with 39 congestive heart failure patients. Heart failure occurs when heart muscles cannot pump enough blood, which can result from narrowed arteries or the long-term effects of high blood pressure.

Research by Roger Hajjar, director of Mount Sinai’s cardiovascular research center and others, led to an approach for treating heart failure that introduces healthy SERCA2a genes into the coronary arteries of patients to help heart cells restore their proper use of calcium. The therapy involves SERCA2a genes that produce SERCA2a enzymes, which help drive the pumping action of the heart, and are often deficient in heart failure patients. Without sufficient SERCA2a enzymes, calcium stays longer in heart cells  than needed, and encourages overgrowth of the number and size of heart cells, leading to an enlarged heart in heart failure patients.

The clinical trial, known as Calcium Up-Regulation by Percutaneous Administration of Gene Therapy In Cardiac Disease or CUPID 1, first tested the therapy for safety, and then compared the treatments to a placebo. The trial was conducted by Celladon that is commercializing the therapy. Hajjar is a scientific co-founder of the company and serves on its advisory board.

The first phase of the trial tested the therapy, called Mydicar by the company, at several dosage levels, which were found to be safe and well-tolerated by heart failure patients.  After 12 months, the group receiving a high-dose of Mydicar had fewer heart problems and deaths from any cause than the group receving the placebo.

After three years, recurrence of heart problems was reduced by 82 percent among patients receiving the therapy. Also after three years, 13 deaths occurred among the patients in the trial, 6 of which among those who received the placebo, 3 each in the low- and mid-dose patients, and 1 in the high-dose patients. Celladon says no safety concerns were reported in the three-year period.

The company is recruiting participants for an intermediate stage clinical trial testing Mydicar against a placebo among heart failure patients. That trial aims to enroll 250 patients in 63 locations.

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