Jyoti Jaiswal, associate professor at George Washington University School of Medicine and Health Sciences and investigator at Children’s Research Institute in Washington, D.C., was awarded an MDA research grant totaling $300,000 over a period of three years to evaluate whether a new anti-inflammatory compound can reduce muscle damage in dysferlinopathies.
Dysferlinopathies are muscle diseases due to mutations in the dysferlin gene, an important muscle repair protein. They include limb-girdle muscular dystrophy 2B and Miyoshi myopathy. Jaiswal is studying patient cells and mouse models to determine whether a new compound, called VBP15, can improve muscle damage.
“Unlike some of the other muscular dystrophies where anti-inflammatory steroidal drugs are beneficial, these drugs are ineffective in dysferlinopathy patients,” Jaiswal says. “At present, there are no drugs available for dysferlinopathy, and the drugs being tested do not simultaneously address the poor muscle cell membrane repair and muscle inflammation — two of the key deficits common to dysferlinopathy patients.”
VBP15 improves the ability of muscle cells to heal damage to their outer membrane and is a potent anti-inflammatory agent that lacks the metabolic side effects associated with use of steroidal anti-inflammatory drugs. It is currently approaching clinical trials for other diseases.
“Establishing the preclinical benefits of VBP15 using dysferlinopathic mice could make this a viable drug-based therapy for dysferlinopathy patients,” Jaiswal says.
Funding for this MDA grant began August 1, 2013.