iPSC-based research in ALS precision medicine

Clinical trials in amyotrophic lateral sclerosis (ALS) are challenged by the lack of pre-clinical models and biomarkers of disease onset and progression. In this issue, Morimoto et al. use induced pluripotent stem cell (iPSC)-derived motor neurons from patients with ALS to study therapeutic mechanisms of ropinirole in a clinical trial and identify treatment responders. Clinical trials in amyotrophic lateral sclerosis (ALS) are challenged by the lack of pre-clinical models and biomarkers of disease onset and progression. In this issue, Morimoto et al. use induced pluripotent stem cell (iPSC)-derived motor neurons from patients with ALS to study therapeutic mechanisms of ropinirole in a clinical trial and identify treatment responders. Phase 1/2a clinical trial in ALS with ropinirole, a drug candidate identified by iPSC drug discoveryMorimoto et al.Cell Stem CellJune 01, 2023In BriefOkano and colleagues conducted a clinical trial to evaluate the safety/efficacy of ropinirole, an anti-ALS drug found via iPSC-based drug discovery that was well tolerated. They identified biomarkers to assess disease progression and demonstrated an effect of ropinirole on the SREBP2-cholesterol synthesis pathway. The trial was small and requires further validation. Full-Text PDF Open Access