Exploiting pluripotent stem cell technology for drug discovery, screening, safety, and toxicology assessments

Abstract

In order for the pharmaceutical industry to maintain a constant flow of novel drugs and therapeutics into the clinic, compounds must be thoroughly validated for safety and efficacy in multiple biological and biochemical systems. Pluripotent stem cells, because of their ability to develop into any cell type in the body and recapitulate human disease, may be an important cellular system to add to the drug development repertoire. This review will discuss some of the benefits of using pluripotent stem cells for drug discovery and safety studies as well as some of the recent applications of stem cells in drug screening studies. We will also address some of the hurdles that need to be overcome in order to make stem cell-based approaches an efficient and effective tool in the quest to produce clinically successful drug compounds.

Keywords: Cardiotoxicity; Disease modeling; Embryonic stem cells; Hepatotoxicity; High content screening; High throughput screening; Induced pluripotent stem cells.