Induced pluripotent stem cells (iPSCs) are increasingly used in the study of disease mechanisms and the development of effective disease-modifying therapies for neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS). Recently, three candidate anti-ALS drugs - ropinirole (ROPI), retigabine, and bosutinib - have been identified in iPSC-based drug screens and are now being evaluated in clinical trials for safety and effectiveness. We review the preclinical data, clinical research design, and rationale for ROPI as an anti-ALS drug candidate compared with those of the other two drugs. We also discuss the use of iPSCs for understanding and monitoring treatment response as well as for new insights into the development of new drugs and therapeutic interventions for major neurodegenerative diseases.

中文翻译：

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Ropinirole，一种使用 iPSC 开发的新型 ALS 候选药物。

诱导多能干细胞 (iPSC) 越来越多地用于疾病机制的研究和神经退行性疾病，包括肌萎缩侧索硬化 (ALS) 的有效疾病修饰疗法的开发。最近，在基于 iPSC 的药物筛选中确定了三种候选抗 ALS 药物——罗匹尼罗 (ROPI)、瑞替加滨和博舒替尼，目前正在临床试验中对其安全性和有效性进行评估。我们回顾了 ROPI 作为抗 ALS 候选药物与其他两种药物的临床前数据、临床研究设计和基本原理。我们还讨论了使用 iPSC 来理解和监测治疗反应，以及对开发新药和治疗主要神经退行性疾病的干预措施的新见解。