Immunotherapy for Advanced Brain Cancer

Immuno-oncology (or immunotherapy) and precision medicine are the newest developments in the treatment of advanced cancer. Immuno-oncology helps to restore the body’s immune system and improves outcomes when administered alone or in combination with chemotherapy.

Immunotherapy offers promising options for treating brain cancer, which is traditionally treated with chemotherapy, radiation therapy, and surgery.

Immunotherapy is a class of treatments that take advantage of a person’s own immune system to help kill cancer cells. There are currently six FDA-approved immunotherapy options for brain and nervous system cancers.

Immunomodulators

  • Dostarlimab (Jemperli): a checkpoint inhibitor that targets the PD-1/PD-L1 pathway; approved for subsets of patients with advanced brain or nervous system cancer that has DNA mismatch repair deficiency (dMMR)
  • Granulocyte-macrophage colony-stimulating factor (GM-CSF): an immunodulatory cytokine; approved in combination with naxitamab-gqgk for a subset of patients with advanced neuroblastoma
  • Pembrolizumab (Keytruda®): a checkpoint inhibitor that targets the PD-1/PD-L1 pathway; approved for subsets of patients with advanced brain or nervous system cancers that have high microsatellite instability (MSI-H), DNA mismatch repair deficiency (dMMR), or high tumor mutational burden (TMB-H)

 

Targeted Antibodies

  • Bevacizumab (Avastin®): a monoclonal antibody that targets the VEGF/VEGFR pathway and inhibits tumor blood vessel growth; approved for advanced glioblastoma
  • Dinutuximab (Unituxin®): a monoclonal antibody that targets the GD2 pathway; approved for first-line treatment of high-risk pediatric neuroblastoma
  • Naxitamab-gqgk (Danyelza®): a monoclonal antibody that targets the GD2 pathway; approved in combination with GM-CSF for a subset of patients with advanced neuroblastoma


Several other immunotherapies are being used to treat different types of brain cancers in clinical trials.
In an attempt to improve the chance of cure, immunotherapies are being tested alone or in combination with chemotherapy in clinical trials.