Evolution of clinical management and therapeutic strategies for diffuse intrinsic pontine glioma

Abstract

Diffuse intrinsic pontine glioma (DIPG) is one of the most lethal pediatric brain tumors, and focal radiotherapy is still the only established standard treatment. However, recent progress in molecular neuro-oncology has substantially changed our understanding of this disease. Histone H3K27 alterations have redefined most classic DIPG cases within the framework of diffuse midline glioma, leading to renewed interest in histopathological diagnosis and biologics-based therapy. Accordingly, the use of stereotactic biopsy; re-irradiation; targeted agents; epigenetic therapies; immunotherapy, including chimeric antigen receptor (CAR) T-cell therapy; and novel drug-delivery approaches, such as convection-enhanced delivery (CED), are being actively investigated. Moreover, as DIPG is a rare condition, registry-based data collection is essential for future clinical trials. This review summarizes evolving disease concepts, current diagnostic strategies, recent therapeutic developments, and future challenges associated with DIPG management in Japan with reference to our institutional experience.