, Volume 14, Issue 2, pp 385–392

Therapeutic Hypothesis Testing With Rodent Brain Tumor Models

  • Derek A. Wainwright
  • Craig M. Horbinski
  • Rintaro Hashizume
  • C. David James

DOI: 10.1007/s13311-017-0523-1

Cite this article as:
Wainwright, D.A., Horbinski, C.M., Hashizume, R. et al. Neurotherapeutics (2017) 14: 385. doi:10.1007/s13311-017-0523-1


The development and application of rodent models for preclinical testing of novel therapeutics and approaches for treating brain tumors has been a mainstay of neuro-oncology preclinical research for decades, and is likely to remain so into the foreseeable future. These models serve as an important point of entry for analyzing the potential efficacy of experimental therapies that are being considered for clinical trial evaluation. Although rodent brain tumor models have seen substantial change, particularly since the introduction of genetically engineered mouse models, certain principles associated with the use of these models for therapeutic testing are enduring, and form the basis for this review. Here we discuss the most common rodent brain tumor models while directing specific attention to their usefulness in preclinical evaluation of experimental therapies. These models include genetically engineered mice that spontaneously or inducibly develop brain tumors; syngeneic rodent models in which cultured tumor cells are engrafted into the same strain of rodent from which they were derived; and patient-derived xenograft models in which human tumor cells are engrafted in immunocompromised rodents. The emphasis of this review is directed to the latter.


Xenograft Syngeneic Immunocompetent vs. immunocompromised Spontaneous vs. engraftment 

Supplementary material

13311_2017_523_MOESM1_ESM.pdf (499 kb)
ESM 1(PDF 499 kb)

Funding information

Funder NameGrant NumberFunding Note
National Institute of Neurological Disorders and Stroke
  • R01NS080619
  • R01NS095642
National Cancer Institute
  • R01CA159467

Copyright information

© The American Society for Experimental NeuroTherapeutics, Inc. 2017

Authors and Affiliations

  1. 1.Department of Neurological SurgeryNorthwestern University Feinberg School of MedicineChicagoUSA
  2. 2.Department of Microbiology and ImmunologyNorthwestern University Feinberg School of MedicineChicagoUSA
  3. 3.Department of Medicine-Hematology/OncologyNorthwestern University Feinberg School of MedicineChicagoUSA
  4. 4.Department of PathologyNorthwestern University Feinberg School of MedicineChicagoUSA
  5. 5.Department of Biochemistry and Molecular GeneticsNorthwestern University Feinberg School of MedicineChicagoUSA

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