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The Muscular Dystrophy Association (MDA) has awarded John Landers, PhD, professor of neurology, a $300,000 research grant to identify new therapeutic targets for amyotrophic lateral sclerosis (ALS). The grant is one of eight totaling more than $2 million awarded by the MDA for ALS research this month.
ALS is a progressive neurodegenerative disease that affects the motor neurons that control muscle cells. People with ALS slowly lose the ability to initiate and control muscle movement, gradually losing function, causing muscles to become weak and eventually nonfunctional. Most cases of ALS are sporadic, meaning there is no known family history of the disease. However, up to 10 percent of cases are familial, meaning the disease is inherited or passed down within a family. Life expectancy is typically three to five years after diagnosis, and there is no cure.
Scientists have identified more than 40 genes that cause familial ALS; some of them have also been found in nonfamilial ALS patients. Still, there is no identified genetic root for 90 percent of ALS cases. Dr. Landers will apply his funding to identifying novel therapeutic targets for both familial and nonfamilial ALS. He will use RNA interference screens, in which RNA molecules bind to and inactivate genes, to explore novel drug targets for ALS.
Additional projects will promote therapy development on multiple fronts. The awarded grants include studies to help provide a better understanding of what causes ALS and pinpoint new drug targets, to develop improved therapeutic strategies, and to ensure that the right tools such as biomarkers are in place to pave the way for successful clinical trials.
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