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John Landers, PhD

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John Landers, PhD

University of Massachusetts

Investigating Pridopidine, a Sigma-1 receptor activator, as a novel therapeutic treatment for ALS
Co-funded with the ALS Association

Within this project, we are developing Pridopidine, a new oral small molecule, for the treatment of Amyotrophic Lateral Sclerosis (ALS). Pridopidine exerts its neuroprotective effects through a protein called sigma-1 receptor (S1R). The S1R regulates several key ALS-relevant cellular processes, and we have data showing that Pridopidine, working through the S1R, could be a powerful new therapeutic for ALS patients. In the following research proposal, we propose key preclinical studies in animals and in-vitro to further establish the potential of Pridopidine as a candidate ALS therapy, based on the known mechanism of action of this drug candidate. Evidence of efficacy in ALS mouse models of different ALS-causing mutations, in human patient-derived cellular models, as well as longitudinal tracking of drug effects on cytoskeletal defects in ALS models, as well as other mechanistic studies, will provide critical data to support a decision by Teva to move this program forward rapidly into the clinic.

 

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Steven Finkbeiner

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Gladstone Institutes, UCSF
Two recently discovered genes that have been associated with both familial and sporadic forms of ALS encode the related proteins TDP43 and FUS cause neuron death in ALS.
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