Using Induced Pluripotent Stem Cells to Discover New Drugs

In recent years, patient-derived induced pluripotent stem cells have been used to recreate in vitro models of human diseases, which have been traditionally studied and modeled in animals. Human induced pluripotent stem cell models seem promising for the discovery of novel drugs suitable for the treatment of human pathologies, such as progressive supranuclear palsy, amyotrophic lateral sclerosis, spinal muscular atrophy, Alzheimer’s disease, and Parkinson’s disease. Some of these drugs are currently entering the first stages of clinical trials, as reported in a recent analysis published in Nature Reviews Drug Discovery. Further efforts are needed to determine whether induced pluripotent stem cell models truly mimic the biological features of human diseases and whether these models can predict drug efficacy in humans. Additionally, improvement of reproducibility and scalability and reduction of production costs are needed if the use of induced pluripotent stem cell models for drug screening projects are to expand.