ArticleNeuro-oncology2025
Locoregional infusion of EGFR806-CAR T cells for recurrent or refractory pediatric CNS tumors: Results of the completed BrainChild02 phase 1 clinical trial.
Article in Neuro-oncology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers, 4 of them syntheses that pooled it.
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
19 citing papers in PubMed, 4 syntheses or guidelines pooled it.
- Efficacy of CAR-T-Cell-Based Immunotherapies in Patients With Glioma: A Systematic Review and Meta-Analysis.Journal of cellular and molecular medicine · 2026Pooled it
- Locoregional and systemic adoptive cellular therapies for pediatric brain tumors: a systematic review of CAR‑T, TCR‑engineered T cells, and NK cell strategies.Neurosurgical review · 2026Pooled it
- Chimeric Antigen Receptor-T Cell Therapy for Pediatric and Young Adult Primary Central Nervous System Tumors: A Systematic Review of Early Phase Clinical Trials with a Focus on Diffuse Midline Glioma.Targeted oncology · 2026Pooled it
- The safety and efficacy of chimeric antigen receptor-T cell therapy in solid malignancies: a systematic review and meta-analysis.Frontiers in immunology · 2026Pooled it
- The DIPG/DMG National Tumor Board: The power of advocacy.Neuro-oncology pediatrics · 2026Article
- Chimeric antigen receptor (CAR)-T cell therapy for solid tumors in pediatric patients: current breakthroughs, dilemmas, and strategies.Experimental hematology & oncology · 2026Review
- Cellular and Immunotherapy for Pediatric Brain Tumors: A Primer.Current oncology (Toronto, Ont.) · 2026Review
- Potent anti-tumor preclinical efficacy of B7-H3 CAR-T cells in H3G34-mutant, Diffuse Hemispheric Glioma.Neuro-oncology · 2026Article
- B7 homolog 3-targeted CAR-T cells secreting EGFR T-cell engagers for improved control of glioblastoma progression.Molecular biomedicine · 2026Article
- Cell therapy for brain tumors: The first 60 years.Cell reports. Medicine · 2026Review
- The development of CAR T cells for patients with CNS malignancies.Nature reviews. Clinical oncology · 2026Review
- Overcoming immunotherapy barriers in pediatric brain tumors: epigenetic strategies.Frontiers in oncology · 2026Review
- CAR T-cells for the treatment of CNS malignancies.Frontiers in immunology · 2026Review
- Optimizing CAR T cell therapy for solid tumours: a clinical perspective.Nature reviews. Clinical oncology · 2025Review
- Physiologically based pharmacokinetic model for CAR-T cell delivery and efficacy in solid tumors.Proceedings of the National Academy of Sciences of the United States of America · 2025Article
- Neurotoxicity in central nervous system tumors treated with CAR T cell therapy: a review.Journal of neuro-oncology · 2025Review
- Transient mRNA CAR T cells targeting GD2 provide dose-adjusted efficacy against diffuse midline glioma and high-grade glioma models.Neuro-oncology · 2025Article
- Physiologically-based pharmacokinetic model for CAR-T cells delivery and efficacy in solid tumors.bioRxiv : the preprint server for biology · 2025Article
- CAR T cell therapy for central nervous system solid tumors: current progress and future directions.Frontiers in immunology · 2025Review
Corrections and comments
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Authors and funding
24 authors.
Funding
Abstract
backgroundRelapsed/refractory pediatric central nervous system (CNS) tumors have a poor prognosis. Epidermal growth factor receptor (EGFR) is commonly overexpressed, but EGFRvIII mutations are uncommon. To target these tumors, we used chimeric antigen receptor (CAR) T cells with a binder based on mAb806 which recognizes ectopically expressed wild-type EGFR and EGFRvIII.
methodsIn this open-label phase 1 clinical trial, patients aged 1-26 years with EGFR + CNS tumors received weekly infusions of 1-2.5 × 107 CAR T cells into the tumor resection bed or the lateral ventricle via an implanted catheter. No lymphodepletion was used.
resultsEleven patients were enrolled. Four (3 with high-grade glioma, 1 with atypical teratoid rhabdoid tumor) were treated and received 5-10 CAR T cell infusions without dose-limiting toxicities. The trial closed prior to reaching planned dose regimens. All treatment-related adverse events were no higher than CTCAE grade 2. The most common were headache and nausea. One patient had a grade 1 seizure, and 3 had new sensory changes, weakness and/or urinary changes (grades 1-2) that were possibly related to CAR T cell infusion. A total of 3 of the 4 treated patients had progressive disease. One patient with spinal cord diffuse midline glioma had progressive peritumoral edema that could not be conclusively attributed to either progression or pseudoprogression and was, therefore, defined as stable disease, followed by a complete response to subsequent chemotherapy.
conclusionsIntracranially infused EGFR806-CAR T cells were tolerable at tested doses, with the best response of stable disease. EGFR is a potentially useful target for cellular therapy against pediatric brain tumors, particularly high-grade gliomas.
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