Pluripotent stem cells for neurodegenerative disease modeling: an expert view on their value to drug discovery

Neurodegenerative diseases have become a major global health concern, posing a huge disease burden on patients and their families. Although there has been rapid progress in the development of therapies, a lack of accurate disease models and efficient drug screening platforms have made achieving a breakthrough difficult. The technology of human-induced pluripotent stem cells (iPSCs) shows better recapitulation of disease pathophysiology and provides a more accessible supply of patient-specific samples compared to other modeling methods. It has been a powerful tool for mechanism exploration and drug development.

This review describes the recent use of human iPSC-derived cells for modeling neurodegenerative disorders and discovering potential drugs.

Model systems based on iPSC-derived cells have created a paradigm shift in drug discovery. Accuracy, consistency, translatability, and cost-effectiveness are the four major focuses of academic and industrial communities to fulfill the potential of iPSC technology for their purposes. It is the art of balance between these four factors to generate efficacious outputs with maximum efficiency. Future studies should persist in refining this technology and promote its application in this field to benefit all the disease-affected population eventually.