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Drug efficacy testing in mice

William Y Kim1, Norman E Sharpless

  • 1The University of North Carolina, Chapel Hill, NC, USA. wykim@med.unc.edu

Current Topics in Microbiology and Immunology
|August 9, 2011
PubMed

Insights

Genetically engineered mouse models (GEMMs) offer a more accurate preclinical cancer drug testing platform than traditional xenografts. Well-characterized GEMMs can improve the selection of effective human cancer therapeutics.

Area of Science:

  • Oncology
  • Translational Medicine
  • Preclinical Research

Background:

  • Traditional drug development relies on in vitro screening and xenografts, which often fail to predict human efficacy.
  • Genetically engineered mouse models (GEMMs) offer improved histopathologic and genetic similarity to human cancers.
  • GEMMs are increasingly utilized for preclinical testing of cancer therapeutics.

Purpose of the Study:

  • To evaluate the suitability of GEMMs for preclinical cancer drug testing.
  • To highlight the advantages and challenges of using GEMMs in drug development.
  • To assess the potential of GEMMs in prioritizing therapeutic agents.

Main Methods:

  • Review of studies utilizing GEMMs for preclinical cancer therapeutic evaluation.
  • Analysis of the histopathologic and genetic characteristics of GEMMs in relation to human cancers.
  • Assessment of the predictive value of GEMMs for drug efficacy.

Main Results:

  • GEMMs demonstrate closer resemblance to human cancer than traditional xenografts.
  • Studies using kinase inhibitors in GEMMs have shown promise.
  • Several advantages and challenges associated with GEMM studies have been identified.

Conclusions:

  • Well-characterized GEMMs are a valuable tool for preclinical cancer research.
  • GEMMs can aid in the prioritization of both established and novel cancer therapeutics.
  • Further development and validation of GEMMs will enhance their role in drug discovery.

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