MDA Awards $750,000 to Help Summit Test New Drug Formulation in Phase 1 Clinical Trial of Potential Treatment for Duchenne Muscular Dystrophy
TUCSON, Ariz., Dec. 19, 2011 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association (MDA ) has awarded $750,000 to Summit PLC , a UK biotechnology company, to continue development of an experimental drug that may become a viable treatment for boys with Duchenne muscular dystrophy (DMD ).
The funding comes from MDA Venture Philanthropy (MVP), a part of MDA's translational research program.
DMD is one of nine types of muscular dystrophy, a group of genetic, degenerative diseases primarily affecting voluntary muscles. Caused by mutations in the gene that makes dystrophin, a protein that normally protects muscle cells and keeps them intact, DMD eventually affects all voluntary muscles, and the heart and breathing muscles. DMD affects 1 in 3,500 boys with an estimated patient population of 50,000 in the developed world.
Summit's SMT C1100 is an experimental compound designed to increase the muscle protein utrophin, which is a naturally-occurring protein that has a similar function to dystrophin. Investigators are confident that if utrophin could be upregulated sufficiently, it could act as a substitute for the missing dystrophin in DMD to help maintain healthy muscles.
"A long-time champion for utrophin upregulation as a promising therapeutic strategy for treating Duchenne muscular dystrophy, MDA is pleased Summit soon will be testing a new formulation of SMT C1100 in a human clinical trial," said Valerie Cwik, M.D. Executive Vice President Research and Medical Director for the Muscular Dystrophy Association.
"This is a vital step toward knowing definitively whether utrophin can successfully stand-in for the missing dystrophin protein that MDA-funded investigators identified as the underlying cause for this form of muscular dystrophy," Cwik added.