Duchenne muscular dystrophy (DMD) is a genetic disease in which patients lack the protein dystrophin, which is critical for stabilizing muscle cell membranes. Without a functional dystrophin protein, muscle cells are susceptible to damage and progressively die. Patients experience progressive muscle weakness starting at an early age, loss of ambulation typically in the first decade of life, eventual respiratory and cardiac failure, and an abbreviated lifespan averaging less than three decades.
Approximately 15,000 to 20,000 boys and young men are living with the disease in the United States1. CAP-1002 has been granted orphan drug designation by the FDA for the treatment of DMD. A growing body of preclinical data supports the use of CAP-1002 for the treatment of DMD. Results presented at the 2014 Annual Meeting of the American Heart Association showed CDCs in preclinical mouse models to be effective in decreasing myocardial fibrosis, improving cardiac function, and inducing cardiomyogenesis, which is the regeneration of heart muscle. Improvements in exercise capacity of unexpected magnitude were also seen after CDC treatment in the preclinical mdx mouse model.
Capricor is conducting the HOPE-2 clinical trial for Capricor’s lead investigational therapy, CAP-1002.
Click here to view a webcast on cardiosphere-derived cells for heart failure in Duchenne muscular dystrophy by Eduardo Marbán, M.D,Ph.D. – Director, Cedars-Sinai Heart Institute
1 Duchenne Muscular Dystrophy United States Prevalence Estimate