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Abstract

EGFRvIII-Targeted CAR-T Cell Therapy: A Systematic Review of Emerging Treatment Strategies for Glioblastoma †

by
Ana C. F. Leite
1,*,
Luigi Gallo
1,
Lavinia H. A. Torres
1,
Mateus G. C. Soares
1,
Samuel C. Silva
1,
Isaac D. S. V. Prado
1 and
Carlos N. Aucélio
2
1
Faculty of Medicine, University of Brasília (UNB), Brasília 70910-900, Brazil
2
Area of Human Morphology, Faculty of Medicine, University of Brasília (UNB), Brasília 70910-900, Brazil
*
Author to whom correspondence should be addressed.
†
Presented at the 6th International Congress on Health Innovation—INOVATEC 2025, Hybrid, 21–23 November 2025.
Proceedings 2026, 137(1), 29; https://doi.org/10.3390/proceedings2026137029
Published: 25 February 2026
(This article belongs to the Proceedings of The 6th International Congress on Health Innovation—INOVATEC 2025)
Versions Notes

Keywords:
GBM; EGFRvIII; CAR-T cells

Glioblastoma (GBM) is the most aggressive primary brain tumor, characterized by marked molecular heterogeneity and resistance to standard therapeutic approaches [1,2,3]. Among the explored molecular targets, the EGFRvIII mutation, present in a subset of patients, stands out for its tumor specificity and immunogenic potential [2,4,5]. This systematic review aimed to critically evaluate the existing evidence on the efficacy, safety, and challenges of EGFRvIII-targeted CAR-T cell therapy in the treatment of glioblastoma. The review aimed to identify current evidence on the efficacy, safety, and challenges of EGFRvIII-targeted CAR-T cell therapy in glioblastoma. A 2014–2024 literature search was conducted in PubMed, Web of Science, and Scopus using “glioblastoma”, “EGFRvIII”, and “CAR-T cells.” Eligible studies included original research, systematic reviews, and clinical trials reporting outcomes, safety, and resistance mechanisms. Seven studies were selected. Preclinical evidence demonstrated that anti-EGFRvIII CAR-T cells are capable of migrating into the brain parenchyma, infiltrating tumor deposits, and selectively lysing neoplastic cells expressing EGFRvIII [2,5,6]. Early-phase clinical trials reported partial responses with transient tumor burden reduction and modest improvements in overall survival, though limited by antigen loss and the development of adaptive resistance [3,7]. Adverse events, generally manageable, included neurotoxicity and cytokine release syndrome of varying severity [2,3,5]. EGFRvIII-directed CAR-T cell therapy represents an innovative and promising strategy against GBM, with encouraging preliminary outcomes in terms of tumor response and safety profile [2,5,7]. Nevertheless, tumor heterogeneity, antigen escape, and immune evasion mechanisms hinder sustained efficacy, highlighting the need for advances in cellular engineering and combinatorial strategies to optimize clinical benefit [1,3,7].

Author Contributions

Conceptualization, A.C.F.L. and C.N.A.; methodology, A.C.F.L.; investigation, A.C.F.L., S.C.S., I.D.S.V.P., L.H.A.T., L.G. and M.G.C.S.; data curation, A.C.F.L.; writing—original draft preparation, A.C.F.L.; writing—review and editing, S.C.S., I.D.S.V.P., L.H.A.T., L.G., M.G.C.S. and C.N.A.; supervision, C.N.A.; project administration, C.N.A. All authors have read and agreed to the published version of the manuscript.

Funding

This research received no external funding.

Institutional Review Board Statement

Not applicable.

Informed Consent Statement

Not applicable.

Data Availability Statement

The data presented in this study are available in Cell & Molecular Immunology at https://doi.org/10.1038/s41423-024-01226-x, reference number [1]; International Journal of Molecular Sciences at https://doi.org/10.3390/ijms25137174, reference number [2]; Science Translational Medicine at https://doi.org/10.1126/scitranslmed.aaa0984, reference number [3]; Oncogene at https://doi.org/10.1038/s41388-017-0045-7, reference number [4]; PLOS ONE at https://doi.org/10.1371/journal.pone.0094281, reference number [5]; Frontiers in Oncology at https://doi.org/10.3389/fonc.2024.1434495, reference number [6]; and Cells at https://doi.org/10.3390/cells13090726, reference number [7] (all accessed on 1 February 2026). These data were derived from resources available in the public domain at the URLs listed above. No new data were generated or analyzed in this study. All data supporting the findings of this work are included within the article and its referenced sources.

Conflicts of Interest

The authors declare no conflicts of interest.

References

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  6. Miao, H.; Choi, B.D.; Suryadevara, C.M.; Sanchez-Perez, L.; Yang, S.; De Leon, G.; Sayour, E.J.; McLendon, R.; Herndon, J.E.; Healy, P.; et al. EGFRvIII-specific chimeric antigen receptor T cells migrate to and kill tumor deposits infiltrating the brain pa-renchyma in an invasive xenograft model of glioblastoma. PLoS ONE 2014, 9, e94281. [Google Scholar] [CrossRef] [PubMed]
  7. O’Rourke, D.M.; Nasrallah, M.P.; Desai, A.; Melenhorst, J.J.; Mansfield, K.; Morrissette, J.J.; Martinez-Lage, M.; Brem, S.; Maloney, E.; Shen, A.; et al. A single dose of peripherally infused EGFRvIII-directed CAR T cells mediates antigen loss and induces adaptive resistance in patients with recurrent glioblastoma. Sci. Transl. Med. 2017, 9, eaaa0984. [Google Scholar] [CrossRef] [PubMed]
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Share and Cite

MDPI and ACS Style

Leite, A.C.F.; Gallo, L.; Torres, L.H.A.; Soares, M.G.C.; Silva, S.C.; Prado, I.D.S.V.; Aucélio, C.N. EGFRvIII-Targeted CAR-T Cell Therapy: A Systematic Review of Emerging Treatment Strategies for Glioblastoma. Proceedings 2026, 137, 29. https://doi.org/10.3390/proceedings2026137029

AMA Style

Leite ACF, Gallo L, Torres LHA, Soares MGC, Silva SC, Prado IDSV, Aucélio CN. EGFRvIII-Targeted CAR-T Cell Therapy: A Systematic Review of Emerging Treatment Strategies for Glioblastoma. Proceedings. 2026; 137(1):29. https://doi.org/10.3390/proceedings2026137029

Chicago/Turabian Style

Leite, Ana C. F., Luigi Gallo, Lavinia H. A. Torres, Mateus G. C. Soares, Samuel C. Silva, Isaac D. S. V. Prado, and Carlos N. Aucélio. 2026. "EGFRvIII-Targeted CAR-T Cell Therapy: A Systematic Review of Emerging Treatment Strategies for Glioblastoma" Proceedings 137, no. 1: 29. https://doi.org/10.3390/proceedings2026137029

APA Style

Leite, A. C. F., Gallo, L., Torres, L. H. A., Soares, M. G. C., Silva, S. C., Prado, I. D. S. V., & Aucélio, C. N. (2026). EGFRvIII-Targeted CAR-T Cell Therapy: A Systematic Review of Emerging Treatment Strategies for Glioblastoma. Proceedings, 137(1), 29. https://doi.org/10.3390/proceedings2026137029

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