July 20, 2011

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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