Currently approved therapies for amyotrophic lateral sclerosis (ALS) — a progressive neurodegenerative disease characterized by loss of motor neurons — exhibit limited effectiveness. By screening a panel of existing drugs in induced pluripotent stem cell (iPSC)-derived motor neurons from patients with familial ALS, Imamura et al. discovered that SRC-ABL inhibitors may protect against motor neuron degeneration. In ALS motor neurons, the SRC-ABL inhibitor bosutinib promoted autophagy, reduced the amount of misfolded superoxide dismutase 1 (SOD1) protein and restored energy homeostasis. In a mouse model of familial ALS, bosutinib delayed disease onset and extended survival.
References
Imamura, K. et al. The Src/c-Abl pathway is a potential therapeutic target in amyotrophic lateral sclerosis. Sci. Transl Med. 9, eaaf3962 (2017)10.1126/scitranslmed.aaf3962
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Crunkhorn, S. SRC-ABL inhibitors protect motor neurons. Nat Rev Drug Discov 16, 530 (2017). https://doi.org/10.1038/nrd.2017.144
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DOI: https://doi.org/10.1038/nrd.2017.144
