Drug efficacy testing in mice

Curr Top Microbiol Immunol. 2012;355:19-38. doi: 10.1007/82_2011_160.


The traditional path of drug development passes from in vitro screening and response assessment to validation of drug efficacy in cell line xenografts. While xenografts have their merits, historically, more often than not, they have not served as an accurate predictor of drug efficacy in humans. The refinement and increased availability of genetically engineered mouse models (GEMMs) of cancer has made GEMMs an attractive avenue for the preclinical testing of therapeutic agents. The histopathologic and genetic resemblance of GEMMs to human cancer are an important measure to evaluate their suitability for pre-clinical studies and a number of studies using kinase inhibitors have now been performed in GEMMs. We have highlighted several of the salient advantages and challenges associated with GEMM studies. Well-characterized GEM models of human cancer should aide in the prioritization of both established and novel therapeutics.

MeSH terms

  • Animals
  • Drug Discovery / methods*
  • Drug Resistance, Neoplasm
  • Drug-Related Side Effects and Adverse Reactions
  • Enzyme Activation
  • Gene Expression Profiling
  • Genes, myc
  • Humans
  • Mice
  • Mice, Transgenic
  • Neoplasms / drug therapy
  • Neoplasms / enzymology
  • Neoplasms / genetics
  • Protein Kinase Inhibitors / chemistry
  • Protein Kinase Inhibitors / pharmacology*
  • Protein Kinases / chemistry*
  • Protein Kinases / genetics
  • Signal Transduction
  • Xenograft Model Antitumor Assays / methods*


  • Protein Kinase Inhibitors
  • Protein Kinases