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Updated: Jul 14, 2026
Generation of CAR T Cells for Adoptive Therapy in the Context of Glioblastoma Standard of Care
Published on: February 16, 2015
Chimeric antigen receptor (CAR)-T-cell therapy for glioblastoma: what can we learn from the early clinical trials? A
Justine Tin Nok Chan1,2, James Henley-Waters1,2, Saeed Kayhanian3,1
1Fitzwilliam College, University of Cambridge, Cambridge, UK.
Background:
Glioblastoma is a malignant brain tumor with poor outcomes. Chimeric antigen receptor-T (CAR-T)-cell therapy is a possible new intervention in solid tumors using T cells engineered with cancer-specific antigens. This approach is challenged by limited T-cell trafficking to solid tumors, the immunosuppressive tumor microenvironment, and glioblastoma-specific uncertainties. Several first-in-human trials have now trialed CAR-T therapy for glioblastoma. We undertake a systematic review of these Phase I trials to draw early lessons about the safety and feasibility of this approach.
Methods:
Systematic review of all published clinical trials using CAR-T therapy for glioblastoma on July 31, 2024, from 5 databases.
Results:
Thirteen published studies of Phase I trials of CAR-T therapy for glioblastoma (n = 128 patients). Six molecular targets were used, most commonly EGFR family (7 studies) and IL13a2 (4 studies). There were 141 severe adverse effects (SAEs) and 2 dose-limiting toxicities. SAEs were most commonly neurological or hematological, and most commonly observed with doses over 1 × 107 cells. Routes of delivery included intravenous, intraventricular, intracavitary, and intratumoral. Several participants across trials demonstrated transient responses, but efficacy across trials was difficult to compare, given heterogeneous reporting of outcomes.
Conclusions:
Several CAR-T strategies have now been trialed preliminarily for glioblastoma. There appears to be a signal of efficacy, with 56 of 128 reported patients demonstrating at least some measure of response. Central delivery of CAR-T cells appears safe with doses up to 2.5 × 107 cells well-tolerated. Subsequent CAR-T trials should standardize reporting of outcomes for better comparison across trials.
Insights
Chimeric antigen receptor-T (CAR-T)-cell therapy shows promise for glioblastoma treatment, with preliminary trials indicating a potential efficacy signal. Further research requires standardized outcome reporting for better comparison of CAR-T cell strategies.
Area of Science:
- Neuro-oncology
- Immunotherapy
- Cellular therapy
Background:
- Glioblastoma is an aggressive brain tumor with limited treatment options.
- Chimeric antigen receptor-T (CAR-T)-cell therapy offers a novel approach by engineering T cells to target cancer-specific antigens.
- Challenges include T-cell trafficking, the immunosuppressive tumor microenvironment, and glioblastoma-specific complexities.
Purpose of the Study:
- To systematically review Phase I clinical trials of CAR-T therapy for glioblastoma.
- To assess the safety and feasibility of CAR-T cell therapy in glioblastoma patients.
- To identify early lessons learned from first-in-human trials.
Main Methods:
- Systematic review of published clinical trials involving CAR-T therapy for glioblastoma.
- Data collected from 5 databases up to July 31, 2024.
- Analysis of 13 Phase I trials with 128 participating patients.
Main Results:
- Six molecular targets were investigated, with EGFR family and IL13a2 being most common.
- 141 severe adverse events (SAEs) and 2 dose-limiting toxicities were reported, predominantly neurological and hematological.
- Transient responses were observed in 56 out of 128 patients, but efficacy comparison was hindered by heterogeneous outcome reporting.
Conclusions:
- Preliminary trials suggest a potential efficacy signal for CAR-T cell therapy in glioblastoma.
- Central delivery of CAR-T cells appears safe at doses up to 2.5 x 10^7 cells.
- Standardized outcome reporting is crucial for future CAR-T cell trials in glioblastoma to enable better comparison and advancement of the therapy.
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