After years of proving itself highly effective in the lab and a large animal study a promising gene therapy to prevent and reverse congestive heart failure is ready for clinical trials now. The therapy was developed, in part, at Thomas Jefferson University's Center for Translational Medicine
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.
"This is the last step you have to take to finish a very long line of research," said Patrick Most, M.D
., adjunct assistant professor of medicine at Thomas Jefferson University, and lead author of the study who now heads the Institute for Molecular and Translational Cardiology at the University of Heidelberg, Germany. "The reversal of cardiac dysfunction 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."
The therapy works by raising diminished levels of the protein S100A1, a calcium-sensing protein in the diseased heart muscle cell, to normal. Previous research suggests this will prevent against heart failure development, particularly in people who have had a heart attack.
According to Dr. Most, "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."
About six million people in the United States have heart failure, and it results in about 300,000 deaths each year.
Work on S100A1 started bench side 15 years ago with Dr. Most and Walter J Koch, Ph.D.
, now director of the Center for Translational Medicine in the Department of Medicine in Jefferson Medical College
of Thomas Jefferson University, who, with his team, have moved the research closer to bedside ever since.