Drug discovery research with iPSC models of neurodegenerative diseases

Abstract

Induced pluripotent stem cells (iPSCs) are widely used in research because they can be used to create models of diseases with the same genomic background as in patients. iPSC-based screening is recognized as a valuable approach in drug discovery research. Additionally, efforts are underway to develop high-quality models for drug discovery and to better integrate translational research with clinical studies. This review focuses on neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease (AD), and provides a broad overview of research using iPSCs, ranging from studies of disease mechanisms to applications in drug discovery. Furthermore, several clinical trials based on iPSC research have been initiated, including those of bosutinib, ropinirole, and ezogabine for ALS, and WVE-004 and BII078 for ALS/FTD. Finally, we also wish to highlight screening studies that incorporate artificial intelligence (AI).