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 melanoma and non-small cell lung cancer. 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). Its use in this study is considered investigational.


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 about this study and to inquire about eligibility, please contact Dr. Antonio Omuro at 212-639-7523.





Disease Status

Relapsed or Refractory