Diffuse intrinsic pontine glioma (DIPG) is a disease of childhood whose abysmal prognosis has remained unchanged for over 50 years. Biologic investigation has been stymied by lack of pretreatment tissue, as biopsy has been reserved for atypical cases. Recent advances in surgical and molecular-analytic techniques have increased the safety and potential utility of biopsy; brainstem biopsy has now been incorporated into several prospective clinical trials. These and other recent efforts have yielded new insights into DIPG molecular pathogenesis, and opened new avenues for investigation.
Trial registration: ClinicalTrials.gov NCT00880061 NCT01130077.