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UC Awarded $1.5 Million for 'Muscle-Wasting Disease' Study
Published January 2009
With the aid of more than $1.5 million in grants from the National Institutes of Health (NIH), a UC structural biologist will continue research that could lead to an effective drug to fight muscle-wasting diseases.
Tom Thompson, PhD, an assistant professor in the department of molecular genetics, biochemistry and microbiology, is studying myostatin, a protein that regulates muscle growth and is naturally found in animals. Antagonizing myostatin—stopping it from working—through protein inhibitors would stimulate muscle growth.
“These inhibitors would greatly benefit patients suffering from many forms of muscle-wasting diseases such as muscular dystrophy, cancer and sarcopenia,” says Thompson.
Sarcopenia is the age-related loss of muscle mass, strength and function. Thompson received a $1.15 million NIH R01 grant in April and a $300,000 Muscular Dystrophy Association grant in July to study myostatin, a member of the TGFbeta family of proteins.
He previously had been awarded a four-year, $260,000 grant from the American Heart Association (AHA). In addition, graduate student Jennifer Cash was awarded a twoyear AHA fellowship to support her work on the research project.
“Myostatin is a very difficult protein to work on, and she has spent countless hours trying to generate enough of it for our structural studies,” says Thompson.
“Our bodies naturally have very effective inhibitors to myostatin, and one of our goals is to determine how these inhibitors interact with myostatin and stop it from working,” Thompson adds.
Thompson uses protein crystallography to look at these proteins on the atomic level. “Understanding the structure allows us to pick apart how these proteins work and how best to inhibit them,” he says.
Thompson also plans to apply this knowledge and work with UC’s Drug Discovery Center to identify small drug-like inhibitors to myostatin.
“We are very excited to have access to an impressive industrialgrade drug discovery facility here at UC,” he says.
Ultimately, Thompson hopes his research will lead to the rational design of myostatin inhibitors that could be used therapeutically for muscle-wasting diseases.