Monoclonal Antibody-Based Treatments for Neuromyelitis Optica Spectrum Disorders: From Bench to Bedside

• 作者: Wenli Zhu Yaling Zhang Yaping Yan Ying Fu Zhen Wang
• 来源期刊: 神经科学通报（英文版）
• 年，卷（期）: 2020,（10）
• 所属期刊栏目: Reviews
• 页码范围: 1213-1224
• 页数: 12页
• 语种: 英文

摘要

Neuromyelitis optica (NMO)/NMO spectrum disorder (NMOSD) is a chronic,recurrent,antibodymediated,inflammatory demyelinating disease of the central nervous system,characterized by optic neuritis and transverse myelitis.The binding of NMO-IgG with astrocytic aquaporin-4 (AQP4) functions directly in the pathogenesis of ＞60％ of NMOSD patients,and causes astrocyte loss,secondary inflammatory infiltration,demyelination,and neuron death,potentially leading to paralysis and blindness.Current treatment options,including immunosuppressive agents,plasma exchange,and B-cell depletion,are based on small retrospective case series and open-label studies.It is noteworthy that monoclonal antibody (mAb) therapy is a better option for autoimmune diseases due to its high efficacy and tolerability.Although the pathophysiological mechanisms of NMOSD remain unknown,increasingly,therapeutic studies have focused on mAbs,which target B cell depletion,complement and inflammation cascade inactivation,bloodbrain-banrier protection,and blockade of NMO-IgG-AQP4 binding.Here,we review the targets,characteristics,mechanisms of action,development,and potential efficacy of mAb trials in NMOSD,including preclinical and experimental investigations.