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Last updated on April 21, 2014 at 9:29 EDT

Gene Therapy Preventing Heart Failure

July 22, 2011

(Ivanhoe Newswire)–A promising gene therapy might just be the next great thing in preventing heart failure. Researchers at Thomas Jefferson University’s Center for Translational Medicine have been working on finding a way to prevent and reverse congestive heart failure. These cardiology researchers demonstrated the feasibility and long term effectiveness as well as safety of S100A1 gene therapy in a large animal model of heart failure.

This therapy works by raising diminished levels of protein S100A1, a calcium sensing protein in the diseased heart muscle cell, to normal levels. Previous research suggests that this will prevent against developing heart failure, particularly in people who have suffered from a heart attack in the past. This is very significant because about six million people in the United States have heart failure which results in about 300,000 deaths per year.

Patrick Most, M.D., assistant professor of medicine at Thomas Jefferson University and lead author of the study started working on S100A1 fifteen years ago. Dr. Most was quoted saying, “We have pursued a completely different path over the years. We have set up a translational pipeline and don’t stick to just one model system. We took it step by step, and did whatever was necessary to go to the next level. We realized early on that a mouse is not a man. You need to design target-tailored translational research strategies and work in human-relevant model systems to take molecular discoveries from bench to bedside.”

In the latest study Dr. Most and Walker J. Koch, Ph.D. and director of the Center for Translational Medicine in the Department of Medicine in Jefferson Medical College of Thomas Jefferson University have recently used a pig model. This test is important because the pig model more closely resembles human physiology, function, and anatomy to determine the effectiveness of this gene therapy. Dr. Koch was quoted saying, “This therapy gets to the core of the disease.”

The therapy is used to make the heart beat stronger and overcome the damage caused by previous heart attacks. Their study is the final set of preclinical data needed to apply for investigational drug status with the United States FDA and go to the first phase of clinical trial. Now the study needs to find industry or private partners to not only help fund the work but recruit eligible patients to enroll in the clinical test.

SOURCE: Science Translational Medicine, July 20, 2011