Gene therapy to fix failing hearts works in pigs and will be tried in humans in 2018

Scientists have been able to reverse heart failure in pigs by delivering a new gene to the heart that makes it better at pumping blood and supplying the body with oxygen. The goal is to reverse heart failure with a single injection. This time around, Dr. Hajjar said, he and his team have reengineered the viral vector to make it more effective at delivering the new gene to the heart.

The gene therapy was safe and reduced heart failure by 25 percent in the left ventricle by 20 percent in the left atrium. Hajjar says most patients with heart failure have problems in the left ventricle. The treatment also reduced the size of the animals’ enlarged hearts by 10 percent.

The pig heart and other organs are about the same size of human organs. The researchers plan to begin enrolling people with advanced congestive heart failure in a clinical trial next year.

About 5.7 million adults in the U.S. have heart failure, and about half of those who are diagnosed die within five years, according to the Centers for Disease Control and Prevention. Heart failure currently can’t be cured; it can only be managed with medications and behavioral changes like quitting smoking, watching your diet, and getting regular physical activity.

A previous human clinical trial failed because it wasn’t getting to enough heart muscle cells. Now the team at Mount Sinai have reengineered the viral vector to make it more effective at delivering the new gene to the heart.

Journal of the American College of Cardiology – Protein Phosphatase Inhibitor-1 Gene Therapy in a Swine Model of Nonischemic Heart Failure.

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