Highlights
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iPSC-based drug discovery is a promising technology for developing novel therapeutics for neurodegenerative diseases lacking useful disease models, such as amyotrophic lateral sclerosis (ALS).
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Ropinirole, retigabine, and bosutinib were identified as candidate therapeutic agents for ALS by the combination of iPSC-based drug discovery and drug repositioning.
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The potential anti-ALS mechanism of ropinirole is independent of antioxidant activity, rescue of mitochondria, reduction of stress granules, and abnormal proteins such as phosphorylated TDP-43 and FUS, and dopamine D2 receptor (D2R) agonism.
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Retigabine inhibits the hyperexcitability of motor neurons in ALS and bosutinib prompts autophagy and reduces abnormal proteins such as SOD-1 and phosphorylated TDP-43 via the Src/c-Abl pathway in motor neurons in ALS.
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Stratification of ALS, personalized medicine strategies, and the identification of common mechanisms with other neurodegenerative diseases are key aspects in the development of ALS therapies.
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