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Gene therapy shows big promise for advanced heart failure

DNA vaccine and Gene therapy techniques are si...
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It’s only a small group of patients, but the results are dramatic. A new gene therapy for advanced heart failure looks really promising.

People can live a long time with heart failure if it’s kept under control. But if it gets to where they can’t keep the water from accumulating beyond safe levels in the body’s tissues, patients begin to feel like hell and experience more frequent cardiac events that put them in the hospital.

Researchers conducted a Phase II trial at Mount Sinai School of Medicine with a gene therapy developed there and found it stabilized or improved cardiac function in people with severe heart failure. The patients who were given a high dose of the therapy, called SERCA2a, benefited clinically (which means they felt better or lived longer) and had significantly fewer cardiovascular hospitalizations. The study appears online in the June 27 issue of the American Heart Association journal Circulation.

Simply put, the SERCA2a therapy consists of delivering an inactive virus that carries medication into cardiac cells. It then stimulates the heart cells to produce an enzyme that helps the heart pump more effectively in people with advanced heart failure.

Quality of life is often just as important as longevity. If you can feel okay and not have to go to the hospital every other week or month, it’s a lot easier to live your life more fully. Advanced heart failure is tough—it’s always exciting to see that science continues to find ways to use the tools of nature to help in relatively non-invasive ways.

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