CAR T-Cell Therapy for Rheumatic Diseases: What Does the Future Hold?

IF 5.4 2区 医学 Q1 IMMUNOLOGY BioDrugs Pub Date : 2025-01-01 Epub Date: 2024-12-29 DOI:10.1007/s40259-024-00692-z
Jérôme Avouac, Marc Scherlinger
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Abstract

Chimeric antigen receptor (CAR) T-cell therapy, initially successful in treating hematological malignancies, is emerging as a potential treatment for autoimmune diseases, including rheumatic conditions. CAR T cells, engineered to target and eliminate autoreactive B cells, offer a novel approach to managing diseases like systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and inflammatory myopathies, where B cells play a pivotal role in disease pathology. Early case reports have demonstrated promising results, with patients achieving significant disease remission, normalization of serological markers, and the ability to discontinue traditional immunosuppressive therapies, which supported the initiation of several clinical trials. However, the application of CAR T-cell therapy in chronic inflammatory rheumatic disorders poses unique challenges, including patient heterogeneity, the risk of adverse effects such as cytokine release syndrome, and the high costs associated with the therapy. Despite these challenges, the potential for CAR T cells to provide long-term remission or even a cure in refractory autoimmune diseases is significant. Ongoing research aims to optimize CAR T-cell constructs and improve safety profiles, paving the way for broader application in rheumatic diseases. If these challenges can be addressed, CAR T-cell therapy could revolutionize the treatment landscape for chronic inflammatory rheumatic disorders, offering new hope for patients with severe, treatment-resistant conditions.

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CAR - t细胞治疗风湿病:未来如何?
嵌合抗原受体(CAR) t细胞疗法最初在治疗血液系统恶性肿瘤方面取得成功,目前正在成为包括风湿病在内的自身免疫性疾病的潜在治疗方法。CAR - T细胞被设计用于靶向和消除自身反应性B细胞,为治疗系统性红斑狼疮(SLE)、系统性硬化症(SSc)和炎性肌病等疾病提供了一种新方法,其中B细胞在疾病病理中起着关键作用。早期病例报告显示了令人鼓舞的结果,患者获得了显着的疾病缓解,血清学标志物正常化,并且能够停止传统的免疫抑制治疗,这支持了几项临床试验的启动。然而,CAR - t细胞疗法在慢性炎症性风湿病中的应用面临着独特的挑战,包括患者异质性,细胞因子释放综合征等不良反应的风险,以及与治疗相关的高成本。尽管存在这些挑战,CAR - T细胞在难治性自身免疫性疾病中提供长期缓解甚至治愈的潜力是显著的。正在进行的研究旨在优化CAR - t细胞结构和提高安全性,为风湿病的更广泛应用铺平道路。如果这些挑战能够得到解决,CAR - t细胞疗法可能会彻底改变慢性炎症性风湿病的治疗前景,为患有严重、治疗难治性疾病的患者带来新的希望。
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来源期刊
BioDrugs
BioDrugs 医学-免疫学
CiteScore
12.60
自引率
2.90%
发文量
50
审稿时长
>12 weeks
期刊介绍: An essential resource for R&D professionals and clinicians with an interest in biologic therapies. BioDrugs covers the development and therapeutic application of biotechnology-based pharmaceuticals and diagnostic products for the treatment of human disease. BioDrugs offers a range of additional enhanced features designed to increase the visibility, readership and educational value of the journal’s content. Each article is accompanied by a Key Points summary, giving a time-efficient overview of the content to a wide readership. Articles may be accompanied by plain language summaries to assist patients, caregivers and others in understanding important medical advances. The journal also provides the option to include various other types of enhanced features including slide sets, videos and animations. All enhanced features are peer reviewed to the same high standard as the article itself. Peer review is conducted using Editorial Manager®, supported by a database of international experts. This database is shared with other Adis journals.
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