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Updated: Sep 5, 2025

Translational Orthotopic Models of Glioblastoma Multiforme
Published on: February 17, 2023
Orthotopic brain tumor models derived from glioblastoma stem-like cells
Katharina M Eyme1, Alessandro Sammarco2, Christian E Badr3
1Department of Neurology, Massachusetts General Hospital, Boston, MA, United States; Department of Neurology, Heidelberg University Hospital, Heidelberg, Germany.
Abstract:
There is an urgency for identifying effective therapies for glioblastoma (GBM), an incurable and lethal primary malignant brain tumor. Patient-derived xenograft mouse models, in which glioma stem cells, which retain the characteristics of the original tumor, are implanted into the brain of immunocompromised mice, represent a well-suited model for studying GBM. Such models are essential for studies involving the tumor microenvironment and for testing experimental therapeutics for brain tumors. In this chapter, we detail various steps for generating an orthotopic brain tumor model in mice. We provide step-by-step guidance for enrichment and expansion of glioma stem cells for surgical specimens, surgical injection of these cells into the brain of immunocompromised mice, as well as monitoring of tumor growth.
Insights
Developing patient-derived xenograft mouse models is crucial for glioblastoma (GBM) research. These models help study the tumor microenvironment and test new therapies for this lethal brain cancer.
Area of Science:
- Neuro-oncology
- Cancer Research
- Animal Models
Background:
- Glioblastoma (GBM) is an aggressive, incurable primary brain tumor.
- Effective therapeutic strategies for GBM are urgently needed.
- Patient-derived xenograft (PDX) mouse models are valuable for GBM research.
Purpose of the Study:
- To provide detailed methodology for establishing orthotopic glioblastoma mouse models.
- To guide researchers in creating reliable models for studying GBM.
- To facilitate preclinical testing of novel GBM therapeutics.
Main Methods:
- Enrichment and expansion of glioma stem cells from surgical specimens.
- Stereotactic injection of glioma stem cells into the brains of immunocompromised mice.
- Monitoring tumor growth and progression in the orthotopic model.
Main Results:
- Successful generation of orthotopic brain tumor models using patient-derived glioma stem cells.
- Demonstrated utility of these models for studying the tumor microenvironment.
- Established a reproducible method for preclinical therapeutic evaluation.
Conclusions:
- Orthotopic patient-derived xenograft mouse models are essential tools for glioblastoma research.
- These models enable comprehensive studies of the tumor microenvironment and therapeutic responses.
- The detailed methodology facilitates the development of effective GBM treatments.
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