ReportsAmyotrophic Lateral Sclerosis

The Src/c-Abl pathway is a potential therapeutic target in amyotrophic lateral sclerosis

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Science Translational Medicine  24 May 2017:
Vol. 9, Issue 391, eaaf3962
DOI: 10.1126/scitranslmed.aaf3962

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A stepping stone to ALS drug discovery

ALS is a heterogeneous motor neuron disease for which there is no treatment and for which a common therapeutic target has yet to be identified. In a new study, Imamura et al. developed a drug screen using motor neurons generated from ALS patient induced pluripotent stem cells (iPSCs). They screened existing drugs and showed that inhibitors of Src/c-Abl kinases promoted autophagy and rescued ALS motor neurons from degeneration. One of the drugs was effective for promoting survival of motor neurons derived from ALS patients with different genetic mutations. The Src/c-Abl pathway may be a potential therapeutic target for developing new drugs to treat ALS.