Utility of Human Stem Cells for Drug Discovery

The pharmaceutical industry continues to struggle to deliver novel and innovative medicines to the market. One of the major challenges in deriving new therapeutics is to more accurately predict the safety and efficacy of the candidate molecule. The current paradigm of drug discovery has several limitations but perhaps the most conspicuous deficiency is the lack of human-based experimental models. The advent of human embryonic stem cells followed by the discovery of induced pluripotent stem (iPS) cells offers unprecedented opportunities for integrating human cellular assays in drug discovery and development. Human iPS cell lines of many diseases have been obtained and iPSC-derived disease affected cells have been utilised for proof-of-concept drug screens to assess efficacy or potential toxicology. The incorporation of iPSC technology thus provides an invaluable opportunity to reduce drug attrition during the process of drug development.