Embryonic stem cells as a source of models for drug discovery

Nat Rev Drug Discov. 2007 Aug;6(8):605-16. doi: 10.1038/nrd2194.


Embryonic stem cells (ESCs) will become a source of models for a wide range of adult differentiated cells, providing that reliable protocols for directed differentiation can be established. Stem-cell technology has the potential to revolutionize drug discovery, making models available for primary screens, secondary pharmacology, safety pharmacology, metabolic profiling and toxicity evaluation. Models of differentiated cells that are derived from mouse ESCs are already in use in drug discovery, and are beginning to find uses in high-throughput screens. Before analogous human models can be obtained in adequate numbers, reliable methods for the expansion of human ESC cultures will be needed. For applications in drug discovery, involving either species, protocols for directed differentiation will need to be robust and affordable. Here, we explore current challenges and future opportunities in relation to the use of stem-cell technology in drug discovery, and address the use of both mouse and human models.

Publication types

  • Review

MeSH terms

  • Animals
  • Cell Culture Techniques / methods
  • Cell Differentiation / drug effects
  • Cell Differentiation / genetics
  • Cell Differentiation / physiology
  • Drug Design*
  • Embryonic Stem Cells / cytology
  • Embryonic Stem Cells / drug effects
  • Embryonic Stem Cells / metabolism*
  • Green Fluorescent Proteins / genetics
  • Green Fluorescent Proteins / metabolism
  • Humans
  • Microscopy, Fluorescence
  • Models, Biological
  • Molecular Structure
  • Myocytes, Cardiac / cytology
  • Myocytes, Cardiac / drug effects
  • Myocytes, Cardiac / metabolism
  • Neurons / cytology
  • Neurons / drug effects
  • Neurons / metabolism
  • Pharmaceutical Preparations / administration & dosage
  • Pharmaceutical Preparations / chemistry
  • Recombinant Fusion Proteins / genetics
  • Recombinant Fusion Proteins / metabolism
  • Technology, Pharmaceutical / methods*


  • Pharmaceutical Preparations
  • Recombinant Fusion Proteins
  • Green Fluorescent Proteins