<scp>CAR</scp> T‐Cell Therapy in Neurology: A Scoping Review of Neuro‐Oncology, Autoimmune Diseases &amp; Neurotoxicity

Chimeric antigen receptor (CAR) T-cell therapy has been investigated in neurological diseases, encompassing both central nervous system malignancies and autoimmune disorders, thereby extending its application beyond hematological cancers. This scoping review evaluates CAR T-cell therapy applications in neurological conditions, assessing therapeutic efficacy, safety profiles, and neurotoxicity management strategies. A literature search across four databases (January 2020-December 2025) identified 33 studies meeting the inclusion criteria, encompassing original and secondary research from international centers. CAR T-cell therapy demonstrated promising efficacy across diverse neurological conditions. In glioblastoma trials, 44% of patients (n = 128) achieved partial or complete clinical/radiographic responses with favorable safety profiles. Moreover, compelling results emerged from neuromyelitis optica spectrum disorder studies, in which 92% of patients (11/12) achieved sustained relapse-free remission over a median follow-up of 5.5 months. Multiple sclerosis, myasthenia gravis, and stiff-person syndrome cases exhibited excellent treatment tolerance without significant immune effector cell-associated neurotoxicity syndrome (ICANS), which is a major concern affecting 27% of patients with hematological malignancies. Overall, CAR T-cell therapy emerges as a novel therapeutic strategy in neurology, encompassing both oncological and autoimmune conditions. Toxicity profiles in neurological CAR T-cell applications differ substantially from those observed in hematologic malignancies, underscoring the need for condition-specific risk assessment frameworks and customized management approaches. Future research should prioritize larger multicenter trials with extended follow-up to establish definitive efficacy and safety profiles in neurological indications.

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Publication Details

Journal
Annals of Clinical and Translational Neurology
Published
2026-04-27
DOI
https://doi.org/10.1002/acn3.70415
Primary Topic
CAR-T cell therapy research
Type
article
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article

CAR T‐Cell Therapy in Neurology: A Scoping Review of Neuro‐Oncology, Autoimmune Diseases & Neurotoxicity

Osama Souied, Ala Dibas, Patricia Tai, Suhair Al‐Ghabeesh et al.
Annals of Clinical and Translational Neurology
CAR-T cell therapy research
article

CAR T‐Cell Therapy in Neurology: A Scoping Review of Neuro‐Oncology, Autoimmune Diseases & Neurotoxicity

Osama Souied, Ala Dibas, Patricia Tai, Suhair Al‐Ghabeesh, Omar Alqaisi, Mohammed Dibas
article en

Abstract

Chimeric antigen receptor (CAR) T-cell therapy has been investigated in neurological diseases, encompassing both central nervous system malignancies and autoimmune disorders, thereby extending its application beyond hematological cancers. This scoping review evaluates CAR T-cell therapy applications in neurological conditions, assessing therapeutic efficacy, safety profiles, and neurotoxicity management strategies. A literature search across four databases (January 2020-December 2025) identified 33 studies meeting the inclusion criteria, encompassing original and secondary research from international centers. CAR T-cell therapy demonstrated promising efficacy across diverse neurological conditions. In glioblastoma trials, 44% of patients (n = 128) achieved partial or complete clinical/radiographic responses with favorable safety profiles. Moreover, compelling results emerged from neuromyelitis optica spectrum disorder studies, in which 92% of patients (11/12) achieved sustained relapse-free remission over a median follow-up of 5.5 months. Multiple sclerosis, myasthenia gravis, and stiff-person syndrome cases exhibited excellent treatment tolerance without significant immune effector cell-associated neurotoxicity syndrome (ICANS), which is a major concern affecting 27% of patients with hematological malignancies. Overall, CAR T-cell therapy emerges as a novel therapeutic strategy in neurology, encompassing both oncological and autoimmune conditions. Toxicity profiles in neurological CAR T-cell applications differ substantially from those observed in hematologic malignancies, underscoring the need for condition-specific risk assessment frameworks and customized management approaches. Future research should prioritize larger multicenter trials with extended follow-up to establish definitive efficacy and safety profiles in neurological indications.

Annals of Clinical and Translational Neurology
Roswell Park Comprehensive Cancer Center (US), Al-Zaytoonah University of Jordan (JO), An-Najah National University (PS), University of Saskatchewan (CA)
Openalex Percentile: Top 6%
CAR-T cell therapy research
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