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B cell-targeting chimeric antigen receptor T cells as an emerging therapy in neuroimmunological diseases. B 细胞靶向嵌合抗原受体 T 细胞作为神经免疫疾病的新兴疗法。
IF 45.5 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2024-06-01 DOI: 10.1016/S1474-4422(24)00140-6
Aiden Haghikia, Georg Schett, Dimitrios Mougiakakos

Background: Neuroimmunology research and development has been marked by substantial advances, particularly in the treatment of neuroimmunological diseases, such as multiple sclerosis, myasthenia gravis, neuromyelitis optica spectrum disorders, and myelin oligodendrocyte glycoprotein antibody disease. With more than 20 drugs approved for multiple sclerosis alone, treatment has become more personalised. The approval of disease-modifying therapies, particularly those targeting B cells, has highlighted the role of immunotherapeutic interventions in the management of these diseases. Despite these successes, challenges remain, particularly for patients who do not respond to conventional therapies, underscoring the need for innovative approaches.

Recent developments: The approval of monoclonal antibodies, such as ocrelizumab and ofatumumab, which target CD20, and inebilizumab, which targets CD19, for the treatment of various neuroimmunological diseases reflects progress in the understanding and management of B-cell activity. However, the limitations of these therapies in halting disease progression or activity in patients with multiple sclerosis or neuromyelitis optica spectrum disorders have prompted the exploration of cell-based therapies, particularly chimeric antigen receptor (CAR) T cells. Initially successful in the treatment of B cell-derived malignancies, CAR T cells offer a novel therapeutic mechanism by directly targeting and eliminating B cells, potentially overcoming the shortcomings of antibody-mediated B cell depletion. WHERE NEXT?: The use of CAR T cells in autoimmune diseases and B cell-driven neuroimmunological diseases shows promise as a targeted and durable option. CAR T cells act autonomously, penetrating deep tissue and effectively depleting B cells, especially in the CNS. Although the therapeutic potential of CAR T cells is substantial, their application faces hurdles such as complex logistics and management of therapy-associated toxic effects. Ongoing and upcoming clinical trials will be crucial in determining the safety, efficacy, and applicability of CAR T cells. As research progresses, CAR T cell therapy has the potential to transform treatment for patients with neuroimmunological diseases. It could offer extended periods of remission and a new standard in the management of autoimmune and neuroimmunological disorders.

背景:神经免疫学的研究与发展取得了长足的进步,尤其是在治疗神经免疫疾病方面,如多发性硬化症、重症肌无力、神经性脊髓炎谱系障碍和髓鞘少突胶质细胞糖蛋白抗体病。仅多发性硬化症就有 20 多种药物获得批准,治疗变得更加个性化。疾病改变疗法,尤其是以 B 细胞为靶点的疗法获得批准,凸显了免疫治疗干预在这些疾病治疗中的作用。尽管取得了这些成功,但挑战依然存在,尤其是对传统疗法无效的患者,这凸显了创新方法的必要性:单克隆抗体(如靶向 CD20 的奥克利珠单抗和 ofatumumab,以及靶向 CD19 的伊奈珠单抗)被批准用于治疗各种神经免疫性疾病,这反映了对 B 细胞活性的理解和管理取得了进展。然而,这些疗法在阻止多发性硬化症或神经性脊髓炎谱系障碍患者的疾病进展或活动方面存在局限性,这促使人们开始探索基于细胞的疗法,特别是嵌合抗原受体(CAR)T 细胞。CAR T 细胞最初成功用于治疗 B 细胞衍生的恶性肿瘤,通过直接靶向和消除 B 细胞提供了一种新的治疗机制,有可能克服抗体介导的 B 细胞耗竭的缺点。CAR T 细胞在自身免疫性疾病和 B 细胞驱动的神经免疫性疾病中的应用表明,CAR T 细胞有望成为一种靶向和持久的选择。CAR T 细胞可自主穿透深层组织,有效消耗 B 细胞,尤其是中枢神经系统中的 B 细胞。虽然 CAR T 细胞的治疗潜力巨大,但其应用也面临着一些障碍,如复杂的物流和治疗相关毒性反应的管理。正在进行和即将进行的临床试验对于确定 CAR T 细胞的安全性、有效性和适用性至关重要。随着研究的深入,CAR T 细胞疗法有可能改变神经免疫疾病患者的治疗方法。它可以延长缓解期,并成为治疗自身免疫性疾病和神经免疫性疾病的新标准。
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引用次数: 0
The prescription of valproate: risk of harm. 丙戊酸钠处方:危害风险。
IF 45.5 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2024-06-01 DOI: 10.1016/S1474-4422(24)00141-8
Fergus Rugg-Gunn, David Hunt
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引用次数: 0
New gene involved in the pathogenesis of Parkinson's disease. 与帕金森病发病机制有关的新基因。
IF 48 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2024-06-01 Epub Date: 2024-04-10 DOI: 10.1016/S1474-4422(24)00166-2
Mathias Toft
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引用次数: 0
Correction to Lancet Neurol 2024; 23: 393-403. Lancet Neurol 2024; 23: 393-403 更正。
IF 48 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2024-06-01 Epub Date: 2024-04-18 DOI: 10.1016/S1474-4422(24)00172-8
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引用次数: 0
Negative findings from trials with NLY01 or deferiprone for Parkinson's disease - Author's reply. 使用 NLY01 或去铁酮治疗帕金森病的试验结果为阴性 - 作者回复。
IF 45.5 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2024-06-01 DOI: 10.1016/S1474-4422(24)00163-7
Andrew McGarry
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引用次数: 0
Correction to Lancet Neurol 2024; 23: 344-81. Lancet Neurol 2024; 23: 344-81 更正。
IF 48 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2024-05-01 Epub Date: 2024-03-18 DOI: 10.1016/S1474-4422(24)00114-5
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引用次数: 0
Complex regional pain syndrome: advances in epidemiology, pathophysiology, diagnosis, and treatment. 复杂区域疼痛综合征:流行病学、病理生理学、诊断和治疗方面的进展。
IF 46.5 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2024-05-01 DOI: 10.1016/S1474-4422(24)00076-0
Michael C Ferraro, Neil E O'Connell, Claudia Sommer, Andreas Goebel, Janet H Bultitude, Aidan G Cashin, G Lorimer Moseley, James H McAuley

Complex regional pain syndrome (CRPS) is a rare pain disorder that usually occurs in a limb after trauma. The features of this disorder include severe pain and sensory, autonomic, motor, and trophic abnormalities. Research from the past decade has offered new insights into CRPS epidemiology, pathophysiology, diagnosis, and treatment. Early identification of individuals at high risk of CRPS is improving, with several risk factors established and some others identified in prospective studies during the past 5 years. Better understanding of the pathophysiological mechanisms of CRPS has led to its classification as a chronic primary pain disorder, and subtypes of CRPS have been updated. Procedures for diagnosis have also been clarified. Although effective treatment of CRPS remains a challenge, evidence-based integrated management approaches provide new opportunities to improve patient care. Further advances in diagnosis and treatment of CRPS will require coordinated, international multicentre initiatives.

复杂性区域疼痛综合征(CRPS)是一种罕见的疼痛疾病,通常发生在创伤后的肢体上。这种疾病的特征包括剧烈疼痛以及感觉、自主神经、运动和营养异常。过去十年的研究为 CRPS 的流行病学、病理生理学、诊断和治疗提供了新的见解。对 CRPS 高危人群的早期识别能力正在提高,过去 5 年中,一些风险因素已经确立,还有一些风险因素在前瞻性研究中被发现。对 CRPS 病理生理学机制的深入了解使其被归类为慢性原发性疼痛疾病,CRPS 的亚型也得到了更新。诊断程序也得到了明确。尽管 CRPS 的有效治疗仍是一项挑战,但循证综合管理方法为改善患者护理提供了新的机遇。CRPS 诊断和治疗的进一步发展需要协调一致的国际多中心倡议。
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引用次数: 0
Insights into the progression of genetic Alzheimer's disease from tau PET. 从 tau PET 透视遗传性阿尔茨海默病的进展。
IF 46.5 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2024-05-01 DOI: 10.1016/S1474-4422(24)00124-8
Agneta Nordberg
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引用次数: 0
Important lessons on healthcare from rare diseases. 从罕见病中汲取医疗保健方面的重要经验。
IF 46.5 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2024-05-01 DOI: 10.1016/S1474-4422(24)00136-4
The Lancet Neurology
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引用次数: 0
Association of Pick's disease with the MAPT H2 haplotype. 皮克病与 MAPT H2 单倍型的关系。
IF 46.5 1区 医学 Q1 CLINICAL NEUROLOGY Pub Date : 2024-05-01 DOI: 10.1016/S1474-4422(24)00123-6
Gaël Nicolas
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引用次数: 0
期刊
Lancet Neurology
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