Testing shows gene therapy shrinks an enlarged heart, improves heart function, human trials to start
Researchers at the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai have successfully tested a powerful gene therapy, delivered directly into the heart, to reverse heart failure in large animal models.
Preclinical testing shows SUMO-1 gene therapy shrinks an enlarged heart, improves heart function, and blood flow. Heart failure affects roughly 5.7 million people in the United States, most commonly the elderly. The condition may require a range of treatment, depending on the severity: doctors may tell patients with moderate cases simply to exercise more or to change their diets, while more severe cases could require implanted devices or even a heart transplant. If successful in human trials, the latest treatment methods would be direct and effective, rather than relying on roundabout lifestyle changes or expensive, intrusive technology.
Dr. Hajjar is the scientific cofounder of the company Celladon, which plans to develop AAV.SERCA2a gene therapy for the treatment of heart failure
Cardio Vascular Diseases (CVD) are the number one cause of death globally: more people die annually from CVDs than from any other cause. An estimated 17.3 million people died from CVDs in 2008, representing 30% of all global deaths. Of these deaths, an estimated 7.3 million were due to coronary heart disease
The new research study findings, published in November 13 issue of Science Translational Medicine, is the final study phase before human clinical trials can begin testing SUMO-1 gene therapy. SUMO-1 is a gene that is "missing in action" in heart failure patients.
"SUMO-1 gene therapy may be one of the first treatments that can actually shrink enlarged hearts and significantly improve a damaged heart's life-sustaining function," says the study's senior investigator Roger J. Hajjar, MD, Director of the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai and the Arthur & Janet C. Ross Professor of Medicine at Mount Sinai. "We are very eager to test this gene therapy in our patients suffering from severe heart failure."
Heart failure remains a leading cause of hospitalization in the elderly. It accounts for about 300,000 deaths each year in the United States. Heart failure occurs when a person's heart is too weak to properly pump and circulate blood throughout their body.