Immunotherapy for brain tumors: understanding early successes and limitations

Expert Rev Neurother. 2018 Mar;18(3):251-259. doi: 10.1080/14737175.2018.1425617. Epub 2018 Jan 11.


Adverse effects and toxicities related to standard treatments for brain tumors significantly reduce patients' quality of life. Although most immunotherapy approaches for solid tumors have not been successful, several early-phase clinical trials are beginning to reveal a potential role for immunotherapy in the treatment of brain tumors. In particular, methods that activate the innate immune system and induce a polyclonal anti-cancer response have demonstrated that brain tumors are susceptible to immune-mediated tumor destruction. Compared with conventional therapies, modulation of the immune system may improve both survivorship and quality of life during and following treatment. Areas covered: An overview of mechanisms of immunotherapy in the context of current treatments for adult and pediatric brain tumors is provided. Results from recent clinical trials will be discussed, focusing on the favorable safety and efficacy profiles of immunotherapeutics. Expert commentary: Although it is too early to judge the long-term safety of immunotherapy for the treatment of patients with brain tumors, early results suggest that these drugs are well-tolerated and may improve survival and quality of life. Importantly, approaches that activate an anti-tumor immune response lay the framework for iterative development of immunotherapies that can reliably treat patients with brain tumors.

Keywords: CAR T cells; DIPG; Immunotherapy; checkpoint blockade; genetically engineered macrophage; glioblastoma; oncolytic virus; vaccine.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Brain Neoplasms / immunology
  • Brain Neoplasms / therapy*
  • Combined Modality Therapy
  • Humans
  • Immunity, Innate
  • Immunotherapy* / adverse effects
  • Immunotherapy* / methods
  • Quality of Life