Chinas Nikolaos A, Alexopoulos Harry
Department of Cell Biology and Biophysics, Faculty of Biology, National and Kapodistrian University of Athens, Athens, Greece.
Front Immunol. 2025 Jul 18;16:1604174. doi: 10.3389/fimmu.2025.1604174. eCollection 2025.
Autoimmunity and autoimmune diseases arise when the immune system erroneously targets self-antigens leading to tissue damage. Consequently, immunomodulatory and mainly immunosuppressive drugs comprise the conventional treatment in conditions such as systemic lupus erythematosus, rheumatoid arthritis and multiple sclerosis. However, many of these agents often fall short of providing a cure and have a limit on symptom management. This underscores the urgent need for even more advanced therapies for patients to constrain progressive disability. Therefore, currently, researchers explore the potential of chimeric antigen receptor (CAR) T-cell therapy for autoimmune diseases considering its success in cancer treatment and specifically in hematological malignancies. This review will examine recent advancements in CAR T-cell therapy for autoimmune disorders, highlighting how CAR T cells can be engineered to precisely target and eliminate autoreactive immune cells that drive these debilitating diseases, particularly those affecting the nervous system such as Multiple sclerosis, Myasthenia gravis, Neuromyelitis optica, Stiff-person syndrome, Autoimmune encephalitis, MOG-antibody disease and Chronic inflammatory demyelinating polyneuropathy. Also, through an analysis of preclinical and clinical data, we will assess the efficacy, safety, potential side effects and limitations of these innovative therapies. Lastly, we will underline the transformative potential of CAR T-cells in Autoimmune Neurology, offering a promising new hope for treatment where conventional therapies have failed.
当免疫系统错误地将自身抗原作为靶标导致组织损伤时，自身免疫和自身免疫性疾病就会出现。因此，免疫调节药物，主要是免疫抑制药物，构成了系统性红斑狼疮、类风湿性关节炎和多发性硬化症等疾病的传统治疗方法。然而，这些药物中的许多往往无法治愈疾病，并且在症状管理方面存在局限性。这凸显了迫切需要为患者提供更先进的疗法以抑制进行性残疾。因此，目前研究人员鉴于嵌合抗原受体（CAR）T细胞疗法在癌症治疗，特别是血液系统恶性肿瘤治疗中的成功，探索其在自身免疫性疾病治疗中的潜力。本综述将探讨CAR T细胞疗法在自身免疫性疾病治疗方面的最新进展，重点介绍如何设计CAR T细胞以精确靶向并消除驱动这些使人衰弱疾病的自身反应性免疫细胞，特别是那些影响神经系统的疾病，如多发性硬化症、重症肌无力、视神经脊髓炎、僵人综合征、自身免疫性脑炎、MOG抗体病和慢性炎症性脱髓鞘性多发性神经病。此外，通过分析临床前和临床数据，我们将评估这些创新疗法的疗效、安全性、潜在副作用和局限性。最后，我们将强调CAR T细胞在自身免疫性神经病学中的变革潜力，为传统疗法失败的治疗提供充满希望的新途径。
