New Gene Therapy to Reverse Heart Failure Ready for Human Clinical Trials

Reporting in the online July 20 issue of Science Translational Medicine, cardiology researchers have demonstrated feasibility, the long-term therapeutic effectiveness and the safety of S100A1 gene therapy in a large animal model of heart failure under conditions approximating a clinical setting.

Cardiac dysfunction (from a heart attack) was reversed in this pre-clinical heart failure model in the pig by restoring S100A1 levels in practically the same setting as in a patient is remarkable and will pave the way for a clinical trial.

About six million people in the United States have heart failure, and it results in about 300,000 deaths each year.

The therapeutic profile of S100A1 is a unique one as it targets and reverses the underlying causes of heart failure: progressive deterioration of contractile performance, electrical instability and energy deprivation

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