Abstract

In this perspective, we congratulate the international efforts to highlight critical challenges in brain tumor research through a recent Consensus Statement. We also illustrate the importance of developing more accurate and clinically relevant early translational in vitro brain tumor models—a perspective given limited emphasis in the Consensus Statement, despite in vitro models being widely used to prioritize candidate therapeutic strategies prior to in vivo studies and subsequent clinical trials. We argue that successful translation of effective novel treatments into the clinic will require investment into the development of more predictive early pre-clinical models. It is in the interest of researchers, clinicians, and ultimately, patients that the most promising therapeutic candidates are identified and translated toward use in the clinic. Highlighting the value of early pre-clinical brain tumor models and debating how such models can be improved is of the utmost importance to the neuro-oncology research community and cancer research more broadly.

Highlights

  • The recent Consensus Statement ‘Challenges to curing primary brain tumors [1]’, produced by a Cancer Research UK convened panel of international experts, represents an important ‘call-to-arms’ to the neuro-oncology research community and should be congratulated for highlighting areas worthy of investment to improve patient outcomes

  • Agreement exists within the cancer research community that more accurate genetically engineered mouse models (GEMMs) and orthotopic patient-derived xenografts (PDXs), along with greater sharing of these resources, will help translate new treatments into the clinic

  • It is important to consider that, in some contexts, these in vivo models may only be as successful as the therapeutic strategies they are used to evaluate

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Summary

Introduction

The recent Consensus Statement ‘Challenges to curing primary brain tumors [1]’, produced by a Cancer Research UK convened panel of international experts, represents an important ‘call-to-arms’ to the neuro-oncology research community and should be congratulated for highlighting areas worthy of investment to improve patient outcomes. Agreement exists within the cancer research community that more accurate genetically engineered mouse models (GEMMs) and orthotopic patient-derived xenografts (PDXs), along with greater sharing of these resources, will help translate new treatments into the clinic. It is important to consider that, in some contexts, these in vivo models may only be as successful as the therapeutic strategies they are used to evaluate. Greater emphasis should be directed toward enhancing and validating early pre-clinical in vitro culture systems used to model brain tumors

The Value of Early Pre-Clinical Models in Brain Tumor Research
A Need to Validate and Contrast In Vitro Models
Toward Early Translational Models of Post-Surgical Disease
Conclusions
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