Multiple Sclerosis Center, National Center of Neurology and Psychiatry, Tokyo, Japan; Department of Immunology, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan.
Neurochem Int. 2019 Nov;130:104315. doi: 10.1016/j.neuint.2018.10.012. Epub 2018 Oct 17.
Neuromyelitis optica (NMO) and neuromyelitis optica spectrum disorder (NMOSD) are autoimmune diseases associated with a disease-specific autoantibody directed against the water channel protein aquaporin-4. Standard immunotherapy, immunosuppressive agents, and corticosteroids can prevent acute attacks and maintain remission in most patients with NMOSD. However, there is a strong need for additional options for patients who are refractory to standard treatments. Emerging therapies targeting specific molecules related to the pathogenicity of NMOSD are currently being developed. The review focuses on improving preventive treatments for NMOSD, including ongoing randomized clinical trials using biological drugs targeting CD19 and CD20 on B cells, interleukin-6, and complement protein C5. The anti-IL-6 receptor monoclonal antibody tocilizumab (TCZ), which can block IL-6 signaling, was shown to be highly effective for refractory patients with NMOSD. Notably, TCZ has marked effects on chronic neuropathic pain and general fatigue in patients refractory to standard medications. TCZ is a promising drug for preventing acute attacks in patients with NMOSD.
视神经脊髓炎(NMO)和视神经脊髓炎谱系疾病(NMOSD)是自身免疫性疾病,与针对水通道蛋白 aquaporin-4 的疾病特异性自身抗体有关。标准免疫疗法、免疫抑制剂和皮质类固醇可以预防 NMOSD 大多数患者的急性发作并维持缓解。然而,对于对标准治疗有抗性的患者,仍然需要更多的选择。目前正在开发针对 NMOSD 发病机制相关特定分子的新兴治疗方法。该综述重点介绍了 NMOSD 的预防治疗的进展,包括正在进行的使用针对 B 细胞上的 CD19 和 CD20、白细胞介素-6 和补体蛋白 C5 的生物药物进行的随机临床试验。抗白细胞介素-6 受体单克隆抗体托珠单抗(TCZ)可阻断白细胞介素-6 信号,对 NMOSD 难治性患者非常有效。值得注意的是,TCZ 对标准药物难治性患者的慢性神经病理性疼痛和全身疲劳有明显作用。TCZ 是预防 NMOSD 患者急性发作的有前途的药物。