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A Novel Therapy for ALS: Allogeneic Schwann Cell Extracellular Vesicles

Goldschmidt, P. J.; Khan, A.; Guest, J. D.; Jimsheleishvili, G.; Graham, P.; Brooks, A. E.; Silvera, R.; Goldschmidt, A. J.; Pearse, D. D.; Dietrich, D. W.; Levi, A. D.

2023-01-23 neurology
10.1101/2023.01.18.23284378 medRxiv
Show abstract

Amyotrophic Lateral Sclerosis (ALS) is a terminal condition with accelerated loss of motor neurons (MN), resulting in the progressive paralysis of affected patients. ALS is either sporadic (90%) or genetically transmitted (10%) and affects cortical (pyramidal) and spinal cord (lower) MN, axons, and respective muscle endplates. ALS research has focused on MN survival, and current FDA-approved therapies provide only small patient survival benefits. This study reports the intravenous (IV) delivery of serial infusions of allogenic Schwann cell-derived extracellular vesicles (SCEV). The recipient had transient clinical stabilization during treatment but deteriorated rapidly during a pause in the infusions. There were no SCEV infusion-related adverse events observed. Allogeneic SCEV appeared safe for IV delivery in this case and may have therapeutic potential.

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