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Nov 14, 2013

Therapeutic Potential of Gene Therapy Shown in Preclinical Study of Heart Failure

  • Scientists at the Cardiovascular Research Center at the Icahn School of Medicine at Mount Sinai report that they have successfully tested a gene therapy, delivered directly into the heart, to reverse heart failure in large animal models.

    The team’s findings (“SUMO-1 Gene Transfer Improves Cardiac Function in a Large-Animal Model of Heart Failure 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, noted Roger J. Hajjar, M.D., director of the research center and the Arthur & Janet C. Ross Professor of Medicine, adding that SUMO-1 is a gene that is “missing in action” in heart failure patients.

    “One month after balloon occlusion of the proximal left anterior descending artery [in a swine model of ischemic heart failure] followed by reperfusion, the animals were randomized to receive either SUMO-1 at two doses, SERCA2a, or both by adeno-associated vector type 1 (AAV1) gene transfer via antegrade coronary infusion,” wrote the investigators. “Control animals received saline infusions. After gene delivery, there was a significant increase in the maximum rate of pressure rise [dP/dt(max)] that was most pronounced in the group that received both SUMO-1 and SERCA2a. The left ventricular ejection fraction (LVEF) improved after high-dose SUMO-1 with or without SERCA2a gene delivery, whereas there was a decline in LVEF in the animals receiving saline. These results support the critical role of SUMO-1 in SERCA2a function and underline the therapeutic potential of SUMO-1 for HF patients.”

    The Mount Sinai group is already engaged in an ongoing gene therapy clinical trial named CUPID, which is in its final phases of testing SERCA2 gene therapy. Phase I and Phase II trial results were positive, demonstrating substantial improvement in clinical events, according to the researchers.

    “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,” continued Dr. Hajjar.



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