amyotrophic lateral sclerosis (ALS) and is now undergoing clinical trial137. In this study, the authors showed the effect of ezogabine on an iPSC model derived from not only ALS patients with mutations in the superoxide dismutase 1 (SOD1) gene, but also ALS patients with mutations in other genes linked to ALS, such as C9orf72 and FUS. It has also been demonstrated that ALS patient-derived iPSC motor neurons initially exhibit a hyper-excitable state, followed by a decrease in excitability138, suggesting that early intervention with ezogabine treatment may be required for the treatment of ALS patients. The observation of similar drug response in different patient groups allowed generalizing the drug responsiveness across ALS patient types. Drug discovery using patient iPSCs derived from multiple genetic forms is of great value, because it allows testing the drug responsiveness in a broad patient population. In contrast, it is hard to analyze the effect of a drug on multiple mouse models simultaneously.
