A Pilot Study of Pembrolizumab Immunotherapy to Treat Recurrent Hypermutated Glioma

Full Title

A Proof-of-Concept, Pilot Study of Pembrolizumab (MK-3475) in Patients with Recurrent Malignant Glioma with a Hypermutator Phenotype


In this study, researchers are evaluating the use of the immunotherapy drug pembrolizumab to treat glioma that has come back despite prior therapy and is “hypermutated.” This means the tumor contains 30 or more different genetic mutations, as detected using genetic sequencing tests. The tumor type can be any glioma, including astrocytoma, oligodendroglioma, and glioblastoma.

Pembrolizumab is already approved to treat several cancers; its use in this study is considered investigational. It works by blocking a molecule called PD-1 that shuts down the immune response. Blocking PD-1 can allow the immune system to detect and attack cancer cells. Pembrolizumab is given intravenously (by vein).


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have glioma that has come back despite prior treatment that included radiation therapy and at least one regimen of chemotherapy.
  • Patients’ tumors must contain 30 or more mutations, as detected using the MSK-IMPACT genetic sequencing tool.
  • At least 4 weeks must pass between the completion of prior therapies and entry into the study.
  • This study is for patients age 18 and older.

For more information and to inquire about eligibility for this study, please contact Dr. Thomas Kaley at 212-639-5122 or the Brain Tumor Hotline at 866-886-9807.





Disease Status

Relapsed or Refractory