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The mighty mouse: genetically engineered mouse models in cancer drug development
Norman E Sharpless1, Ronald A Depinho
1Departments of Medicine and Genetics, The Lineberger Comprehensive Cancer Center, The University of North Carolina, Chapel Hill, North Carolina 27599-7295, USA. nes@med.unc.edu
Nature Reviews. Drug Discovery
|August 18, 2006
Summary
Standard preclinical cancer drug testing models like xenografts have limitations. Novel genetically engineered mouse models offer better human cancer mimicry, potentially improving drug development success rates and clinical translation.
Area of Science:
- Oncology
- Translational Medicine
- Preclinical Drug Development
Background:
- Standard preclinical models, such as xenograft mouse models, face significant limitations in accurately predicting human cancer drug efficacy.
- These limitations hinder the translation of basic cancer research discoveries into effective clinical treatments.
Purpose of the Study:
- To discuss the established uses and limitations of xenograft mouse models in cancer drug development.
- To explore the potential of novel genetically engineered mouse models (GEMMs) that better recapitulate human cancer evolution.
- To identify opportunities and challenges in applying GEMMs for improved cancer drug discovery.
Main Methods:
- Review of existing literature on xenograft models for cancer drug evaluation.
- Description of the characteristics and advantages of genetically engineered mouse models.
- Analysis of the application of GEMMs in key stages of drug development.
Main Results:
- Xenograft models have inherent limitations in mimicking human cancer's genetic and biological complexity.
- Genetically engineered mouse models offer a more faithful representation of human cancer progression and heterogeneity.
- GEMMs show promise in target validation, response assessment, pharmacodynamic studies, resistance modeling, and toxicity evaluation.
Conclusions:
- Greater utilization of genetically engineered mouse models can significantly enhance the success rate of anticancer drug development.
- Addressing the challenges associated with GEMMs is crucial for their widespread adoption.
- Improved preclinical models are essential for bridging the gap between basic research and clinical benefit in oncology.
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