Gavriilaki Maria, Kimiskidis Vasilios K, Gavriilaki Eleni
Postgraduate Course, School of Medicine, Aristotle University of Thessaloniki, 54124 Thessaloniki, Greece.
Laboratory of Clinical Neurophysiology, AHEPA Hospital, Aristotle University of Thessaloniki, 54621 Thessaloniki, Greece.
Pharmaceuticals (Basel). 2020 Oct 26;13(11):341. doi: 10.3390/ph13110341.
Precision medicine has emerged as a central element of healthcare science. Complement, a component of innate immunity known for centuries, has been implicated in the pathophysiology of numerous incurable neurological diseases, emerging as a potential therapeutic target and predictive biomarker. In parallel, the innovative application of the first complement inhibitor in clinical practice as an approved treatment of myasthenia gravis (MG) and neuromyelitis optica spectrum disorders (NMOSD) related with specific antibodies raised hope for the implementation of personalized therapies in detrimental neurological diseases. A thorough literature search was conducted through May 2020 at MEDLINE, EMBASE, Cochrane Library and ClinicalTrials.gov databases based on medical terms (MeSH)" complement system proteins" and "neurologic disease". Complement's role in pathophysiology, monitoring of disease activity and therapy has been investigated in MG, multiple sclerosis, NMOSD, spinal muscular atrophy, amyotrophic lateral sclerosis, Parkinson, Alzheimer, Huntington disease, Guillain-Barré syndrome, chronic inflammatory demyelinating polyneuropathy, stroke, and epilepsy. Given the complexity of complement diagnostics and therapeutics, this state-of-the-art review aims to provide a brief description of the complement system for the neurologist, an overview of novel complement inhibitors and updates of complement studies in a wide range of neurological disorders.
精准医学已成为医疗科学的核心要素。补体,作为一种数百年来为人所知的固有免疫成分,已被认为与众多无法治愈的神经疾病的病理生理学有关,成为一个潜在的治疗靶点和预测生物标志物。与此同时,首款补体抑制剂在临床实践中作为治疗重症肌无力(MG)和与特定抗体相关的视神经脊髓炎谱系障碍(NMOSD)的获批疗法的创新性应用,为在有害神经疾病中实施个性化治疗带来了希望。基于医学主题词(MeSH)“补体系统蛋白”和“神经疾病”,于2020年5月前在MEDLINE、EMBASE、Cochrane图书馆和ClinicalTrials.gov数据库进行了全面的文献检索。补体在MG、多发性硬化症、NMOSD、脊髓性肌萎缩症、肌萎缩侧索硬化症、帕金森病、阿尔茨海默病、亨廷顿病、吉兰 - 巴雷综合征、慢性炎症性脱髓鞘性多发性神经病、中风和癫痫的病理生理学、疾病活动监测及治疗方面的作用已得到研究。鉴于补体诊断和治疗的复杂性,本前沿综述旨在为神经科医生简要介绍补体系统,概述新型补体抑制剂,并更新在广泛神经疾病中的补体研究情况。