Genetically Engineered Mouse Models of Brain Cancer and the Promise of Preclinical Testing

Brain Pathol. 2009 Jan;19(1):132-43. doi: 10.1111/j.1750-3639.2008.00234.x.


Recent improvements in the understanding of brain tumor biology have opened the door to a number of rational therapeutic strategies targeting distinct oncogenic pathways. The successful translation of such "designer drugs" to clinical application depends heavily on effective and expeditious screening methods in relevant disease models. By recapitulating both the underlying genetics and the characteristic tumor-stroma microenvironment of brain cancer, genetically engineered mouse models (GEMMs) may offer distinct advantages over cell culture and xenograft systems in the preclinical testing of promising therapies. This review focuses on recently developed GEMMs for both glioma and medulloblastoma, and discusses their potential use in preclinical trials. Examples showcasing the use of GEMMs in the testing of molecularly targeted therapeutics are given, and relevant topics, such as stem cell biology, in vivo imaging technology and radiotherapy, are also addressed.

Publication types

  • Review

MeSH terms

  • Animals
  • Brain Neoplasms / drug therapy
  • Brain Neoplasms / genetics
  • Brain Neoplasms / pathology*
  • Disease Models, Animal*
  • Drug Evaluation, Preclinical / methods*
  • Genetic Engineering / methods*
  • Glioma / drug therapy
  • Glioma / genetics
  • Glioma / pathology
  • Humans
  • Medulloblastoma / drug therapy
  • Medulloblastoma / genetics
  • Medulloblastoma / pathology
  • Mice
  • Mice, Transgenic