Progress and prospects in targeted therapy for autoimmune encephalitis

HONG Zhen, CHENG Peng

Journal of Neurology and Neurorehabilitation ›› 2025, Vol. 21 ›› Issue (6) : 433-444.

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Journal of Neurology and Neurorehabilitation ›› 2025, Vol. 21 ›› Issue (6) : 433-444. DOI: 10.12022/jnnr.2025-0278
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Progress and prospects in targeted therapy for autoimmune encephalitis

  • HONG Zhen, CHENG Peng
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Abstract

Autoimmune encephalitis (AE) is a group of inflammatory disorders caused by immune-mediated attacks on the central nervous system, with common clinical manifestations including psychiatric and behavioral abnormalities, cognitive and motor impairments and epileptic seizures. Conventional first-line therapies mainly rely on corticosteroids, intravenous immunoglobulin, and plasma exchange. However, a subset of patients exhibits suboptimal responses or high relapse rates, underscoring the urgent need for the development of novel therapeutic strategies. In recent years, targeted therapeutic approaches have advanced rapidly in the field of AE, primarily focusing on key immune components such as B cells, T cells, the complement cascade, and key inflammatory cytokines. Anti-CD20 monoclonal antibodies have demonstrated promising efficacy in refractory AE. Moreover, emerging agents such as ocrelizumab, epratuzumab, telitacicept, and daratumumab have provided refined strategies for the precise depletion of B cells and plasma cells. In parallel, cytokine-targeting biologics, Janus kinase inhibitors, and the proteasome inhibitor bortezomib have shown potential value for modulating aberrant immune responses. In addition, chimeric antigen receptor (CAR)-T cell immunotherapy is gradually emerging as an important frontier in exploratory research. Looking ahead, by integrating neurorestorative strategies with artificial intelligence-assisted diagnostic and therapeutic decision-making, targeted therapies are expected to drive AE management from traditional empirical immunosuppression toward a new era of precision intervention, thereby enabling more efficient and personalized disease management.

Key words

Autoimmune encephalitis / Pathogenesis / First-line therapy / Targeted therapy

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HONG Zhen, CHENG Peng. Progress and prospects in targeted therapy for autoimmune encephalitis[J]. Journal of Neurology and Neurorehabilitation. 2025, 21(6): 433-444 https://doi.org/10.12022/jnnr.2025-0278
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