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Immune checkpoint inhibitors in GBM
Eudocia Q Lee1,2,3
1Center for Neuro-Oncology, Dana-Farber Cancer Institute, 450 Brookline Avenue, Boston, MA, 02215, USA. eqlee@partners.org.
Journal of Neuro-Oncology
|October 2, 2021
Summary
Immune checkpoint inhibitors show limited survival benefit for glioblastoma (GBM) patients in recent trials. Further research is needed to identify biomarkers and combination therapies for this brain cancer.
Area of Science:
- Neuro-oncology
- Immunotherapy
- Cancer Research
Background:
- Glioblastoma (GBM) remains a challenging brain cancer with poor prognosis.
- Immune checkpoint inhibitors (ICIs) have revolutionized cancer treatment but show limited efficacy in GBM.
- Understanding brain immunology and resistance mechanisms is crucial for improving GBM treatment.
Purpose of the Study:
- To review recent advancements in immune checkpoint inhibitor (ICI) therapy for glioblastoma (GBM).
- To cover updates in brain immunology, clinical trials, resistance mechanisms, and biomarkers for GBM patients receiving ICIs.
Main Methods:
- A comprehensive literature search of PubMed was conducted for relevant articles on ICI therapy in GBM.
- Clinicaltrials.gov was searched to identify ongoing and completed clinical trials related to ICIs in GBM.
Main Results:
- Randomized phase 2 and 3 clinical trials of ICIs in GBM (newly diagnosed or recurrent) have not yet shown a significant survival benefit.
- A preliminary study suggested potential benefits of neoadjuvant/adjuvant pembrolizumab, requiring further validation.
- Response rates to ICIs in GBM patients are generally low.
Conclusions:
- Only a small fraction of glioblastoma patients benefit from current immune checkpoint inhibitor therapies.
- Identifying predictive biomarkers of response is essential for patient selection.
- Developing rational combination strategies with ICIs is necessary to enhance treatment efficacy in GBM.

