Induced pluripotent stem cells ‐based disease modeling, drug screening, clinical trials, and reverse translational research for amyotrophic lateral sclerosis

Over the past 15  years since the advent of induced pluripotent stem cell (iPSC) technology, interest in its medical applications has steadily increased. We have established iPSCs from patients suffering from various intractable neurological diseases, including amyotrophic lateral sclerosis (ALS). By inducing thei r differentiation into MNs, we analyzed their pathological conditions, carried out drug discovery screenings, and identified Ropinirole hydrochloride as a potential treatment for ALS. Subsequent clinical trials were conducted with this drug. Drawing from these findings, we embarked on reverse transl ational research. This review article provides a comprehensive summary of our findings from this series of studies. AbstractIt has been more than 10  years since the hopes for disease modeling and drug discovery using induced pluripotent stem cell (iPSC) technology boomed. Recently, clinical trials have been conducted with drugs identified using this technology, and some promising results have been reported. For amyotrophic lateral sclerosis ( ALS), a devastating neurodegenerative disease, several groups have identified candidate drugs, ezogabine (retigabine), bosutinib, and ropinirole, using iPSCs-based drug discovery, and clinical trials using these drugs have been conducted, yielding interesting results. In our previous study, an iPSCs -based drug repurposing approach was utilized to show the potential of ropinirole hydrochloride (ROPI) in reducing ALS-specific pa...
Source: Journal of Neurochemistry - Category: Neuroscience Authors: Tags: REVIEW Source Type: research