Chris Weihl, assistant professor of neurology at Washington University School of Medicine in St. Louis, was awarded an MDA grant totaling $397,064 over a period of three years. The funds will help support Weihl’s research into a process called autophagy in skeletal muscle. Data gleaned from Weihl’s studies may be applicable to a number of neuromuscular disorders including amyotrophic lateral sclerosis (ALS) and the muscular dystrophies; Weihl and colleagues will conduct their studies on a mouse model of myofibrillar myopathy (MFM).

Autophagy, which means "self-digestion," is a cellular cleanup and garbage-disposal system. Cells use it to degrade and destroy abnormal cellular or protein components that otherwise could lead to toxicity and cell death.

One hallmark in a number of neuromuscular diseases is the presence of protein clumps called aggregates or inclusions in affected tissues. It’s unknown whether facilitating the clearance or degradation of these inclusions is beneficial.

Weihl and colleagues plan to test FDA-approved drugs reported to enhance autophagy to determine whether protein degradation via autophagy is protective. The group will test the drugs in a newly developed mouse model of myofibrillar myopathy.

“These studies will answer the question of whether enhancing autophagy in protein-aggregate disorders is protective,” Weihl said.

Funding for this MDA grant began February 1, 2012.