Gene therapy set to become 'next great thing in heart failure'

Washington, July 21 (ANI): A promising gene therapy to prevent and reverse heart failure is ready for clinical trials, scientists at Thomas Jefferson University's Center for Translational Medicine have reported.

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.

"This therapy gets to the core of the disease," said Dr. Walter J Koch, who received the "Outstanding Investigator Award" for 2011 by the International Society for Heart Research for his work in heart failure gene therapy.

"They are not just beta blockers or ancillary drugs, which only block the damage. This therapy makes the heart beat stronger and overcomes the damage from previous heart attacks. It's the next great thing in heart failure," he added.

The study was recently published in the journal Science Translational Medicine. (ANI)