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Updated: Aug 9, 2026
Testing Cancer Immunotherapeutics in a Humanized Mouse Model Bearing Human Tumors
Published on: December 16, 2022
Harnessing preclinical mouse models to inform human clinical cancer trials
David H Gutmann1, Kim Hunter-Schaedle, Kevin M Shannon
1Department of Neurology, Washington University School of Medicine, St. Louis, Missouri 63110, USA. gutmannd@neuro.wustl.edu
Abstract:
The urgent need for better cancer treatments has stimulated interest in employing small-animal models to evaluate potential drug therapies. Robust mouse models of many human cancers have been generated using sophisticated technologies for engineering germ-line mutations. As we enter into an age of targeted therapeutics, these strains provide novel platforms for validating new anticancer drugs, assessing therapeutic index, identifying surrogate markers of tumor progression, and defining epigenetic and environmental influences on tumorigenesis.
Insights
Genetically engineered mouse models offer advanced platforms for evaluating new cancer drug therapies and understanding tumor development. These models are crucial for advancing targeted cancer treatments and personalized medicine approaches.
Area of Science:
- Oncology
- Genetics
- Pharmacology
Background:
- The development of effective cancer treatments is a critical unmet medical need.
- Small-animal models are essential tools for preclinical drug evaluation.
- Genetically engineered mouse models (GEMMs) offer robust platforms for cancer research.
Purpose of the Study:
- To highlight the utility of GEMMs in evaluating novel anticancer drug therapies.
- To underscore the role of GEMMs in advancing targeted therapeutics.
- To explore the application of GEMMs in understanding cancer biology and treatment response.
Main Methods:
- Generation of GEMMs using sophisticated germ-line mutation engineering technologies.
- Utilizing GEMMs for preclinical validation of anticancer drugs.
- Employing GEMMs to assess therapeutic index and identify biomarkers.
Main Results:
- GEMMs provide powerful systems for validating new anticancer drugs.
- These models facilitate the assessment of drug efficacy and safety.
- GEMMs aid in identifying surrogate markers for tumor progression and treatment response.
Conclusions:
- Genetically engineered mouse models are indispensable for the advancement of cancer drug discovery.
- These models are crucial for the development of targeted cancer therapeutics.
- GEMMs enable a deeper understanding of tumorigenesis, including epigenetic and environmental factors.
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