Towards Advanced iPSC-based Drug Development for Neurodegenerative Disease

Neurodegenerative diseases (NDDs) are a heterogeneous group of diseases that are characterized by the progressive loss of neurons leading to motor, sensory, and/or cognitive defects. Currently, NDDs are not curable and treatment focuses on alleviating symptoms and halting disease progression. Phenotypic heterogeneity between individual NDD patients, lack of robust biomarkers, the limited translational potential of experimental models, and other factors have hampered drug development for the treatment of NDDs. This review summarizes and discusses the use of induced pluripotent stem cell (iPSC) approaches for improving drug discovery and testing. It highlights challenges associated with iPSC model ing and also discusses innovative approaches such as brain organoids and microfluidic-based technology which will improve drug development for NDDs.

Automated summary

Neurodegenerative diseases (NDDs) are a group of heterogeneous diseases characterized by the progressive loss of neurons leading to various defects. Current treatments focus on alleviating symptoms and halting disease progression, but drug development for NDDs faces challenges such as phenotypic heterogeneity and lack of robust biomarkers. Innovative approaches like induced pluripotent stem cell (iPSC) methods and brain organoids are being explored to improve drug development for NDDs.