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Pediatric CNS Tumor Model Development

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.

Seattle Children’s and the Vitanza Lab are honored to be considered a Center of Excellence by the Swifty Foundation and their Gift from a Child initiative. For some patients and families, the donation of tumor after death is a step in honoring their journey and continuing to fight cancer. While this option is not the right choice for every family, this generous act can be important for many families. Our program partners with Gift from a Child to provide a destination for tumors to be compassionately processed and shared with the national and international research community so that they can contribute to better understanding of these tumors and to the identification of future, curative treatments.