<scp>CAR</scp> T‐Cell Therapy in Neurology: A Scoping Review of Neuro‐Oncology, Autoimmune Diseases & 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.
Authors
- Osama Souied (ORCID: https://orcid.org/0000-0001-5730-0088)
- Ala Dibas (ORCID: https://orcid.org/0009-0003-0073-1313)
- Patricia Tai (ORCID: https://orcid.org/0000-0001-7700-776X)
- Suhair Al‐Ghabeesh (ORCID: https://orcid.org/0000-0002-8345-0140)
- Omar Alqaisi (ORCID: https://orcid.org/0009-0006-9760-651X)
- Mohammed Dibas (ORCID: https://orcid.org/0009-0000-7324-264X)
Institutions
- Roswell Park Comprehensive Cancer Center (US)
- Al-Zaytoonah University of Jordan (JO)
- An-Najah National University (PS)
- University of Saskatchewan (CA)
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
- Field-Weighted Citation Impact
- 0.00