Stem Cells in Drug Development

Abstract

In the last 20 years, costs of pharmaceutical development and evaluation have grown dramatically. Numerous failed clinical trials indicate that drug development and testing in animal models and standard cell lines is not reliably predictive for human efficacy and safety. The derivation of human pluripotent stem cells (PSCs), which are relatively normal, grow indefinitely in culture and can be used to generate all cell types of the body holds great promise for faster, cheaper and safer development of drugs. The capability to generate human PSCs from patients with diseases also allows for the derivation of disease models that will enable the elucidation of disease mechanisms while developing targeted drugs. Furthermore, PSCs will enable the development of personalized medicine once large banks of cell lines representing the population have been generated and characterized. As of today, only one validated test based on stem cells exists, accordingly this chapter focusses on the current research into development of PSCs for drug development and evaluation.