The mighty mouse: genetically engineered mouse models in cancer drug development

Nat Rev Drug Discov. 2006 Sep;5(9):741-54. doi: 10.1038/nrd2110. Epub 2006 Aug 18.


Deficiencies in the standard preclinical methods for evaluating potential anticancer drugs,such as xenograft mouse models, have been highlighted as a key obstacle in the translation of the major advances in basic cancer research into meaningful clinical benefits. In this article, we discuss the established uses and limitations of xenograft mouse models for cancer drug development, and then describe the opportunities and challenges in the application of novel genetically engineered mouse models that more faithfully mimic the genetic and biological evolution of human cancers. Greater use of such models in target validation, assessment of tumour response, investigation of pharmacodynamic markers of drug action, modelling resistance and understanding toxicity has the potential to markedly improve the success of cancer drug development.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Antineoplastic Agents / therapeutic use*
  • Drug Design*
  • Drug Screening Assays, Antitumor / methods
  • Humans
  • Mice
  • Mice, Transgenic*
  • Neoplasms, Experimental / drug therapy*
  • Neoplasms, Experimental / genetics
  • Reproducibility of Results


  • Antineoplastic Agents