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Updated: Jul 19, 2026

Development and Maintenance of a Preclinical Patient Derived Tumor Xenograft Model for the Investigation of Novel Anti-Cancer Therapies
Published on: September 30, 2016
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.
Abstract:
The use of genetically engineered cancer-prone mice as relevant surrogates for patients during the development of pertinent clinical applications is an unproven expectation that awaits direct demonstration. Despite the generally disappointing findings using tumor xenografts and certain early transgenic cancer models to predict therapeutic efficacy in patients, the dramatic progress of mouse models in recent years engenders optimism that the newest generation of mouse models will provide a higher standard of predictive utility in the process of drug development.
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.
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