Sunitha Rangaraju, a postdoctoral research scientist at the Scripps Research Institute in La Jolla, Calif., was awarded an MDA development grant totaling $180,000 over a period of three years to determine whether compounds that slow certain aspects of aging may be therapeutic in amyotrophic lateral sclerosis (ALS).
The biggest known risk factor for ALS is advancing age, suggesting that the processes that contribute to aging also may contribute to ALS. This led Rangaraju and colleagues to hypothesize that compounds that slow the aging process might be valuable as drugs against ALS. To that end, their lab screened more than 89,000 molecules for those that delay aging and extend life span in C. elegans, a small worm that is widely used to study aging.
That screen turned up more than 100 molecules that extend worm life span. Rangaraju is now testing those compounds in a worm model of ALS to see if they also contribute to longevity in the context of the disease.
The worm research model of ALS shares important characteristics with human ALS, including “protein aggregation, movement defects, reduction of neuron-to-muscle signals and a shorter life span,” Rangaraju says. She already has identified several compounds that extend life span in these worms, and now will test others, and determine how the most promising ones influence protein aggregation or other aspects of ALS. She then plans to test the most promising molecules in mouse models of ALS, the next step in drug development.
“In the field of ALS, great progress has been made in developing animal models of the disease and unraveling disease mechanisms,” says Rangaraju. “These findings allow us to test novel strategies to develop therapeutic molecules to ameliorate the disease.”
Funding for this MDA grant began Feb. 1, 2013.
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