Local delivery methods of therapeutic agents in the treatment of diffuse intrinsic brainstem gliomas

• Prognosis for patients diagnosed with diffuse brainstem tumors remains dismal because of the limited treatment options currently available.
• Convection enhanced delivery of therapeutic compounds is safe and feasible.
• Interstitial continuous infusion therapy, neural stem cells, and intranasal delivery of CNS tumor-specific compounds are under investigation.

Brainstem gliomas comprise 10–20% of all pediatric central nervous system (CNS) tumors and diffuse intrinsic pontine gliomas (DIPGs) account for the majority of these lesions. DIPG is a rapidly progressive disease with almost universally fatal outcomes and a median survival less than 12 months. Current standard-of-care treatment for DIPG includes radiation therapy, but its long-term survival effects are still under debate. Clinical trials investigating the efficacy of systemic administration of various therapeutic agents have been associated with disappointing outcomes. Recent efforts have focused on improvements in chemotherapeutic agents employed and in methods of localized and targeted drug delivery. This review provides an update on current preclinical and clinical studies investigating treatment options for brainstem gliomas.