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Treatment-Naïve Biopsy-Derived DIPG Models

Diffuse intrinsic pontine glioma (DIPG) is a universally fatal tumor of the brainstem, most commonly affecting young children. Due to its location, surgical resection is not achievable, but consideration of a biopsy has become standard practice at children's hospitals with the appropriate neurosurgical expertise. While the decision to obtain a biopsy should be directed by the presence of atypical radiographic features that call the diagnosis of DIPG into question or the requirement of biopsy tissue for clinical trial enrollment, once this precious tissue is available its use for research should be considered.

The majority of DIPG and diffuse midline glioma, H3 K27M-mutant (DMG) models are autopsy-derived or genetically engineered, each of which are incredibly important but have limitations for translational studies. The use of biopsy tissue for laboratory model development provides an opportunity to create unique model systems from tumors that have yet to receive any treatment, and thus replicate the initial disease. Given developments in the ability to analyze DIPG tumor tissue to deepen biological understanding of this disease and develop new therapies for treatment, together with the safety of brainstem biopsy, we have developed a detailed laboratory protocol for the generation of treatment-naïve biopsy-derived DIPG/DMG models.

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