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New ALS Research and Moonshot Era

Richard Smith

A trailblazer in the field of ALS research and therapy development, Richard Smith, MD, director of the Center for Neurologic Study, and a member of the ALS Network’s scientific review committee, is advocating a change in the way drugs are developed for ALS and similar diseases. To accelerate the discovery process, he’s advocating for funding moonshot ideas that are approved within months if not weeks. To advance this concept, the Center for Neurologic Study is contributing $1.5M to the ALS Network’s therapeutic discovery efforts.

Smith’s most recent research, published in the European Journal of Neurology and highlighted in Nature, is as novel as his success in developing drugs for ALS. Smith, along with Amprion Pharmaceuticals and colleagues worldwide, discovered a subgroup of ALS patients in which self-replicating toxic proteins are detectable and likely contribute to disease origination and progression. 

Next, in collaboration with the ALS Network, Smith plans to implement a natural history study to discern if the course of ALS is different in those in which the self-replicating protein is detected. The ALS Network is uniquely positioned to help facilitate studies like Smith proposes. Relationships with each of the specialized ALS clinics across California and Hawaii, where people receive multidisciplinary care, will help reach the largest ALS patient population in the nation.

Learn more about the ALS Network’s research program here.

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