Diffuse intrinsic pontine glioma (DIPG) is a tumor of the brainstem, specifically in the pons, accounting for 10–20% of all of central nervous system (CNS) tumors in children. Unfortunately, DIPG is the leading cause of death in children with CNS cancers. Clinical interventions trying to effectively treat children with DIPG have failed despite 40 years of clinical trials. The critical location of these tumors eliminates extensive surgical resection as an option. Radiation therapy (RT) is the standard of care, and although it improves the clinical symptoms of most patients, the results are temporary, with tumor progression typically occurring months post radiation. Given the dismal prognosis associated with this disease and the challenge to find chemotherapeutic agents, especially molecularly targeted drugs that improve the survival of the patients, there is a strong incentive to move new treatments forward into clinical trials. The more effective treatment would potentially involve combinatory therapeutic regimens with new epigenetic drugs that can offer synergistic benefits and potentially increase therapeutic efficacy. The increasing knowledge of genomic, epigenomic, and proteomic characteristics of DIPG is opening doors to new therapeutic avenues and provides hope and promise for this devastating childhood cancer.
Part of the book: Neurooncology