Ground-Breakthrough: Gene Therapy That Cures Heart Failure

Finally, a gene therapy that is able to effectively treat heart failure has been discovered. At Mount Sinai School of Medicine, a group of researchers used SERCA2a (a product by MYDICAR®) to generate the expression of an enzyme that promotes a failing heart to pump better.

In phase II of the study, it was shown that injecting SERCA2a responded positively in minimising the severity of heart failure. Early this week, this information was presented at the Heart Failure congress of the European society of Cardiology in Berlin. Jill Klaman, MD, Associate Professor, Medicine, Cardiology, director of the Cardiomyopathy Program at Mount Sinai School of Medicine said that SERCA2a seems to be a “safe and Effective” means to treat patients with advanced heart failure.

Moreover, Klaman also highlighted that there is an insignificant availability of treatment for this patient population with heart failure. It appears that SERCA2a will be a potential solution for the group.

A CUPID trial was conducted. There was a total of 39 patients who suffered from severe heart failure that was enrolled to the randomized, double-blind, placebo-controlled study. The study was geared to test the efficacy and safety of SERCA2a. The patients were randomly picked to receive SERCA2a gene delivery in one of three doses or placebo. They were then evaluated over a period of six months. Moreover, this treatment is directly delivered into a patient’s heart through contemporary outpatient catheterization procedures.

CUPID stands for: (Calcium Up-regulation by Percutaneous administration of gene therapy In cardiac Disease

It was found that patient having been injected with SERCA2a experienced an improvement in their heart functions and decrease severity of heart failure. The patients did also benefit from a less frequent occurrence of cardiovascular events. The study also proved that SERCA2a was safe to consume. There were no signs of adverse events, laboratory abnormalities or disease related issues.


The gene SERCA2a was created 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 at Mount Sinai School of Medicine. The team did early in 1999 identify the potentials of SERCA2a as a gene therapy treatment.

Members of Mount Sinai Heart said that they remain committed in providing further ground breaking therapies to the surface. Moreover, they are also interested in continuing and seeing how crucial this treatment will be in the future.

General Heart Facts

The U.S Centers for Disease Control & Prevention estimates that there are around 5.8 million Americans that have heart failures. There are also 670,000 new cases diagnosed per annum. Studies have also shown that out of every 5 individuals diagnosed having heart failure, one of them dies within a year post to the diagnosis. The cost in America related to heart failure will account for $39.2 billion in 2010. This includes the cost of medication, health care and lost in productivity.

Moreover, heart failure is treated with device therapies and medicines but have no real cure available yet. The symptoms associated with heart failure are breathing problems, feeling of tiredness, and having angles, legs or feet swollen.

Sources: Mount Sinai and E-science News

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