SMA Research Boosted by 2 MDA Grants Totaling $510K

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by Steve Bryson PhD |

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The Muscular Dystrophy Association (MDA) has awarded $17 million among 70 new grants toward neuromuscular disease research, including $510,000 to two projects focused on spinal muscular atrophy (SMA).

“The Muscular Dystrophy Association continues to fund the most innovative research that will lead to cures for a range of neuromuscular diseases,” Sharon Hesterlee, PhD, MDA’s chief research officer, said in an association press release. “We have already seen our investment pay off with the first effective neuromuscular disease therapies, and these grantees are pushing the envelope even further in diseases once thought incurable.”

Nandhini Sivakumar, PhD, of Columbia University Medical Center, in New York, was awarded a $210,000 development grant to explore new ways of boosting the activity of motor neurons — the specialized nerve cells that control voluntary muscle movement and that are lost in SMA.

Motor neurons that directly activate individual muscles are regulated by neurons known as pre-motor neurons, but the underlying molecular mechanisms of this process are unknown.

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Sivakumar will use cell-based and animal models of SMA to investigate the molecular and cellular function of potassium channels in motor neurons. Such channels, which transport potassium ions across cell membranes, are known to be essential for the repetitive activation of motor neurons and subsequent muscle contractions.

By analyzing the effect of molecules that modify the activity of potassium channels, the researchers hope to uncover the molecular events leading to a boost in motor neuron function.

“Our study has the potential to reveal salient principles in the restoration of neuronal function in spinal muscular atrophy and offer novel therapeutic avenues,” Sivakumar wrote in the project description.

In addition, a $300,000 MDA grant was awarded to Rashmi Kothary, PhD, at the Ottawa Hospital Research Institute, in Canada, for his research exploring the contribution of non-neuronal defects, specifically metabolic abnormalities, to SMA.

Kothary and his team previously showed that a deficiency in the SMN protein, which causes motor neuron loss in SMA, also leads to defects in fatty molecule metabolism, suggesting a potential role in disease development.

They also found that children with SMA are more susceptible to liver steatosis, or the build-up of too much fat in the liver.

Kothary will explore the events that trigger liver steatosis in SMA and evaluate whether the liver is an important player in SMA development.

“The research will have important implications in the care and disease management of patients with SMA,” Kothary wrote in the project description.

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The two grants were among 70 awarded this year by the MDA toward research in neuromuscular diseases, such as amyotrophic lateral sclerosis, Duchenne muscular dystrophy, Charcot-Marie-Tooth disease, and myasthenia gravis.

“The Muscular Dystrophy Association has consistently been a leader in advancing treatments for individuals with neuromuscular disease,” said Donald Wood, PhD, MDA’s president and CEO, said. “Patients are now living longer, more independent lives, due in large part to the more than $1 billion we have invested in research over the past [70-plus] years.”

“We are pleased to invest an additional $17 million during this grant cycle to continue to fund the research that will lead to increasing advancements in neuromuscular disease treatments and cures,” Wood added.

Each grant will advance neuromuscular disease research in different ways, such as revealing underlying disease mechanisms, identifying new therapeutic targets, building infrastructure that will accelerate clinical trials, and providing early career support to young researchers.

“The organization’s model of funding research across many neuromuscular diseases means findings from one disease often enable progress in others, maximizing the speed at which progress can be made,” the association stated in the release.

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