The use of targeted mouse models for preclinical testing of novel cancer therapeutics

Kenneth P Olive1, David A Tuveson

  • 1Abramson Family Cancer Research Institute, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Insights

Genetically engineered mouse models show promise for predicting cancer drug efficacy in patients. While past models were disappointing, newer generations offer greater predictive utility for clinical applications.

Area of Science:

  • Oncology
  • Translational Medicine
  • Genetics

Background:

  • Genetically engineered mouse models (GEMMs) are increasingly utilized in preclinical research.
  • Previous models like tumor xenografts and early transgenic models have shown limited success in predicting patient therapeutic responses.
  • Advancements in mouse model development offer renewed optimism for their predictive capabilities.

Purpose of the Study:

  • To evaluate the predictive utility of genetically engineered cancer-prone mice in clinical applications.
  • To assess the potential of novel mouse models in drug development.

Main Methods:

  • Review of existing literature on tumor xenografts and transgenic cancer models.
  • Analysis of recent advancements in the development of genetically engineered mouse models.
  • Assessment of the potential for these models to serve as surrogates for patients in clinical trials.

Main Results:

  • Historically, tumor xenografts and early transgenic models have yielded disappointing results in predicting therapeutic efficacy.
  • Recent progress in mouse model technology suggests a higher standard of predictive utility is achievable.
  • The expectation of GEMMs as patient surrogates requires further demonstration.

Conclusions:

  • Newer generations of genetically engineered mouse models hold significant promise for improving drug development.
  • Further validation is needed to confirm the predictive power of these advanced models in clinical settings.
  • Optimism exists for enhanced translational value of mouse models in oncology research.