A common chemical used in the manufacturing and pharmaceutical industries can repair damage to cardiac muscle cell membranes and prevent heart failure in mice with the genetic mutation that causes Duchenne muscular dystrophy, according to scientists at the University of Michigan Medical School.
The mutation in the dystrophin gene causes the progressive deterioration of skeletal muscles seen in people with MD. But the mutation affects cardiac muscle, too. Many people with Duchenne muscular dystrophy die in their 20s from heart failure caused by cardiomyopathy, a gradual weakening of the heart muscle. Heart failure is the second leading cause of death in DMD.
The chemical sealant that protected hearts in dystrophic mice from damage is called poloxamer 188. According to Joseph M. Metzger, Ph.D., the U-M scientist who directed the research, poloxamer 188 can insert itself into small holes in cell membranes just like “a finger in a dike”.
The U-M study will be published July 17 in Nature as an advance online publication.
“If issues of dosing and long-term safety can be resolved, our research suggests that poloxamer 188 could be a new therapeutic agent for preventing or limiting progressive damage to the hearts of patients with muscular dystrophy,” Metzger, a professor of molecular and integrative physiology and of internal medicine in the U-M Medical School, says.