Related Experiment Video
Updated: May 13, 2026

Transplantation of Zebrafish Pediatric Brain Tumors into Immune-competent Hosts for Long-term Study of Tumor Cell Behavior and Drug Response
Published on: May 17, 2017
Human tumor xenografts and mouse models of human tumors: re-discovering the models
1Genzyme Corporation, 49 New York Avenue, Framingham, MA 01701-9322, USA +1 508 271 2843 ; +1 508 620 1203 ; Beverly.Teicher@Genzyme.com.
Background:
Human tumor xenografts constitute the major efficacy and tumor biology models for cancer drug discovery. Gene expression and protein expression technologies have improved understanding of the malignant diseases represented by human tumor lines. Genetically engineered mouse (GEM) models of cancer are improving and allow assessment of immune system-mediated therapeutics.
Objective:
The current brief review discusses the status of human tumor xenografts, GEM models and normal tissue assays used in cancer drug discovery.
Results:
The Preclinical Pediatric Tumor Program consortium with 47 molecularly well-characterized pediatric tumor xenografts is leading the field. Individual laboratory studies show no correlation between culture and in vivo characteristics of some human tumor lines. The TRAMP and RIP1TAG2 GEM models have been used for efficacy studies. Recent GEM models often have two mutated genes leading to greater genetic instability. Host normal cells are important contributors in malignant disease. While convenient and frequent subcutaneous tumor implantation does not provide an optimal microenvironment for modeling clinical disease. Orthoptopic tumor implantation is gaining in use due to the availability of labeled tumor sub-lines and non-invasive imaging techniques.
Conclusion:
The science associated with human tumor xenografts and GEM cancer models is not static. The application of gene and protein expression technologies, the wider availability of non-invasive imaging and improved genetic engineering are allowing improved tumor modeling. However, as more is learned about current models, it is becoming clear that there is a shortage of models to cover the broad spectrum of human malignant disease.
Insights
Human tumor xenografts and genetically engineered mouse (GEM) models are crucial for cancer drug discovery. Advances in technology improve these models, yet a broader range of models is needed to represent diverse human cancers.
Area of Science:
- Oncology
- Cancer Research
- Drug Discovery
Background:
- Human tumor xenografts and genetically engineered mouse (GEM) models are essential tools in cancer drug discovery.
- Gene and protein expression technologies enhance understanding of cancer biology.
- GEM models are increasingly utilized for evaluating immunotherapies.
Purpose of the Study:
- To review the current status of human tumor xenografts, GEM models, and normal tissue assays in cancer drug discovery.
- To highlight advancements and limitations in preclinical cancer modeling.
Main Methods:
- Review of existing literature on human tumor xenografts and GEM models.
- Discussion of technological advancements like gene/protein expression analysis and non-invasive imaging.
- Evaluation of different implantation techniques (subcutaneous vs. orthotopic).
Main Results:
- The Preclinical Pediatric Tumor Program features 47 well-characterized pediatric tumor xenografts.
- Some studies indicate a lack of correlation between in vitro and in vivo characteristics of certain human tumor lines.
- Orthotopic implantation is gaining favor over subcutaneous methods for better clinical relevance.
- Recent GEM models incorporate multiple gene mutations for increased genetic instability.
Conclusions:
- Cancer modeling using human tumor xenografts and GEMs is continuously evolving with new technologies.
- Improved genetic engineering and imaging techniques enhance tumor modeling capabilities.
- There is a significant need for more diverse preclinical models to cover the spectrum of human cancers.
Related Concept Videos
Mouse Models of Cancer Study
The development of transgenic, knockout, and knock-in mice has led to an exponential increase in their use as model organisms in research,...
Mouse Models of Cancer Study
The development of transgenic, knockout, and knock-in mice has led to an exponential increase in their use as model organisms in research,...

