Beverly Hills, California-based Capricor Therapeutics, Inc. has just announced plans to explore the potential of using one of their leading pipeline products indicated for heart disease, for the treatment of complications from an extremely rare muscle-wasting disease called, Duchenne Muscular Dystrophy (DMD). This specialized biotechnology company has been focused on ways to prevent and treat cardiovascular problems, and product candidate CAP-1002 may answer some questions on DMD-related cardiomyopathies as well..
The company will be structuring an appropriate clinical program for DMD utilizing CAP-1002. This formulation makes use of experimental, allogeneic cell therapy obtained from a donor’s cardiac tissue, which is then instilled directly into a recipients affected coronary arteries via catheterization. At the moment, it is still undergoing Phase II clinical trials for adult patients diagnosed with cardiac ischemia, but soon the company’s researchers will be exploring how it can help solve cardiac muscle weakening commonly seen in DMD patients. While DMD is quite rare, it has been noted to be more common in young males below 6 years old, and many do not live past their mid-20s. Today, DMD is estimated to affect almost 20,000 boys in the US, and roughly 275,000 in the world.
Capricor’s CEO, Linda Marbán, Ph.D., explains the cardiosphere-derived cells (CDCs) have been tested for their safety and efficacy in reducing tissue scarring in ischemic heart disease. Exploring this treatment’s effects on DMD patients will contribute greatly to what is known about CDC technology, possible applications and patient therapeutic responses.
Eduardo Marbán, M.D., Ph.D., the company’s Scientific Advisory Board Chairman and the current Director of the Cedars-Sinai Heart Institute, previously led a study entitled, “Heart-derived Cell Therapy for Duchenne Cardiomyopathy: Cardiosphere-derived Cells and their Exosomes Improve Function, Restore Mitochondrial Integrity and Reverse Degenerative Changes in the Hearts of Mdx Mice”, that will soon serve as a foundation for Capricor’s DMD clinical program. His findings are scheduled to be presented at the upcoming Late Breaking Basic Science Posters and Reception during the American Heart Association’s Scientific Sessions in South Hall A2 of McCormick Place, Chicago on Monday, November 17th, 2014 from 4:00 p.m. to 6:00 p.m.
Research and development for DMD keeps optimistic because of small miracles like in the case of little Jenson Whitehouse, who was diagnosed with DMD when he was one. Despite being told he would never be able to walk, he beat the odds, got up on his walker, and moved about the family living room.