Towards Immunotherapy for Pediatric Brain Tumors

Trends Immunol. 2019 Aug;40(8):748-761. doi: 10.1016/ Epub 2019 Jun 19.


Pediatric brain tumors are the leading cause of childhood cancer-related death. Immunotherapy is a powerful new approach for treating some refractory cancers; applying this 'fourth pillar' of cancer treatment to pediatric brain tumors is an exciting but challenging prospect. This review offers new perspectives on moving towards successful immunotherapy for pediatric brain tumors, focusing on pediatric high-grade glioma (HGG), a subgroup with universally poor outcomes. We cover chimeric antigen receptor T cell (CAR-T) therapy, vaccine therapy, and checkpoint inhibition in this context, and focus on the need for intimately understanding the growing brain and its immune system. We highlight the challenges associated with the application of immunotherapy in pediatric neuro-oncology, as well as the tissue-specific challenges to be overcome, to achieve improved outcomes.

Keywords: CAR-T cells; DIPG; DMG; glioblastoma; glioma; immunotherapy; pediatric brain tumor; vaccine; virus.

Publication types

  • Review

MeSH terms

  • Age Factors
  • Animals
  • Antineoplastic Agents, Immunological
  • Biomarkers, Tumor
  • Brain Neoplasms / etiology
  • Brain Neoplasms / metabolism
  • Brain Neoplasms / pathology
  • Brain Neoplasms / therapy*
  • Child
  • Disease Management
  • Disease Susceptibility
  • Humans
  • Immunotherapy* / methods
  • Immunotherapy, Adoptive / methods
  • Molecular Targeted Therapy
  • T-Lymphocyte Subsets / immunology
  • T-Lymphocyte Subsets / metabolism


  • Antineoplastic Agents, Immunological
  • Biomarkers, Tumor