MDA awarded Professor Bernard Jasmin, vice-dean of research at the University of Ottawa in Canada, a grant totaling $360,000 to study the impact of small molecules called "exercise mimetics" in Duchenne muscular dystrophy (DMD).
One approach to counteract the effects of the loss of the muscle protein dystrophin in DMD involves increasing levels of a similar structural protein called utrophin. Studies performed in a widely used mouse model of DMD, the mdx mouse, have shown that increased utrophin expression can compensate for the lack of dystrophin and ameliorate symptoms of the disease.
Because utrophin activity normally is low and focused only in certain regions of the muscle, Jasmin's laboratory looks for ways to increase levels of the protein in DMD muscles.
"Our first preclinical study showed that treatment of mdx mice with an exercise mimetic known as GW501516 resulted in an increase in utrophin expression, improved muscle function and improved pathological symptoms of the disease," Jasmin said.
His current work builds on the previous study results, with plans for continued testing of GW501516 in the mdx mouse model of DMD, and expansion of the research to include testing of other molecules including AICAR, beta-GPA and resveratrol.
Because of the possible effects of exercise mimetics on utrophin in DMD-affected muscles, the small molecules hold potential for dramatic improvement in quality of life for individuals with the disease. GW501516 currently is under development to treat obesity by the pharmaceutical company GlaxoSmithKline, which could help speed its development for therapeutic use in people with DMD (see DMD, BMD Research: Utrophin from Obesity Drug?).
"MDA funding has allowed us to take a relatively basic molecular observation and perform research that eventually could result in development of novel therapeutic approaches for treatment of people with DMD," Jasmin said.
Funding for this MDA grant began August 1, 2010.
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