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The results of a clinical study found that a new kind of gene therapy reversed heart failure in large animals. The therapy involves sending the SUMO-1 gene directly to the heart where it will work to improve heart function. If the SUMO-1 gene therapy is approved by the U.S. Food and Drug Administration (FDA), human clinical trials could start very soon.

"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," said 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."

For this study, Hajjar and fellow researchers discovered that the SUMO-1 gene was missing in heart failure patients. Prior to this study, the research team had identified another gene that was missing in these patients. In that study, Hajjar conducted a trial named CUPID in which his team administered the SERCA2 gene in a virus through the coronary arteries and directly into the heart using the cardiac catheterization procedure. The SERCA2 gene is responsible for producing an enzyme that helps pump calcium out of cells properly. When the body is going through heart failure, SERCA2 becomes dysfunctional, which forces the heart to work harder. The heart then grows larger.

The results of phase 1 and phase 2a of CUPID were successful. But once the researchers discovered that the SUMO-1 gene was missing as well, they conducted three different gene therapy trials. In the first one, the researchers administered the SUMO-1 gene alone. In the second one, they performed the SERCA2 gene therapy alone and in the last one, they used a combination of both therapies. The therapies only require a one-time dose.

The researchers found that SUMO-1 gene therapy and the combination therapy were the most effective in reversing heart failure. Both therapies led to increased heart contractions, improved blood flow and reduced heart volumes. The researchers hope that they can start testing these therapies on human patients.

"These new study findings support the critical role SUMO-1 plays for SERCA2 function, and underlie the therapeutic potential of SUMO-1 gene replacement therapy for heart failure patients," reported Hajjar.

The study was published in Science Translational Medicine.