REVIEW article
Front. Immunol.
Sec. Cancer Immunity and Immunotherapy
Volume 16 - 2025 | doi: 10.3389/fimmu.2025.1600403
This article is part of the Research TopicAdvances in Immune Cell Engineering for Treating Cancers and Other DiseasesView all 4 articles
CAR T Cell Therapy for Central Nervous System Solid Tumors: Current Progress and Future Directions
Provisionally accepted- 1Karolinska Institutet (KI), Solna, Sweden
- 2Department of Oncology-Pathology, Karolinska Institutet (KI), Stockholm, Stockholm, Sweden
- 3Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Moscow Oblast, Russia
- 4Institute of Bioorganic Chemistry (RAS), Moscow, Moscow Oblast, Russia
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Central nervous system (CNS) tumors are the second most common type of cancer in children and remain the leading cause of mortality in pediatric oncology. For patients with high-risk CNS tumors, standard treatments often prove ineffective, with survival rates being less than 10%. Hence, there is an urgent need to develop alternative treatment strategies for this patient population. Globally, numerous clinical trials are actively investigating a range of novel therapeutic approaches, from pharmacological and immunological therapies to physical modalities targeting the tumor. Among these emerging therapies, CAR T cell therapy has shown great promise, with the first objective clinical responses already reported. This review aims to evaluate the current landscape of CAR T cell therapy for pediatric CNS tumors, focusing on clinical efficacy, toxicity profiles of systemic and locoregional delivery, antigen heterogeneity, and key challenges in clinical implementation. We provide a comprehensive analysis of reported clinical trials, including not only CAR T cell studies but also investigations involving tumor-infiltrating lymphocytes (TILs), NK and lymphokine-activated killer (LAK) cells, offering a broader perspective on immunotherapeutic approaches for CNS malignancies.
Keywords: CNS tumors, Glioma, DMG, dipg, Glioblastoma, CAR T, CAR T solid tumors, brain cancer
Received: 26 Mar 2025; Accepted: 23 Jul 2025.
Copyright: © 2025 Kaminskiy, Maschan, Degtyarev and Stepanov. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Michael Maschan, Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Moscow Oblast, Russia
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