阿尔茨海默病靶向Aβ疾病修饰治疗:曙光初现  

Anti-amyloid-βdisease-modifying therapies for Alzheimer′s disease:the dawn of a new era

作　　者：黄钰媛 郁金泰 Huang Yuyuan;Yu Jintai(Department of Neurology,Huashan Hospital,Fudan University,National Center for Neurological Diseases,Shanghai 200040,China)

机构地区：[1]复旦大学附属华山医院神经内科、国家神经疾病医学中心,上海200040

出　　处：《中华神经科杂志》2023年第9期959-964,共6页Chinese Journal of Neurology

基　　金：科技创新2030-“脑科学与类脑研究”重大项目(2022ZD0211600)。

摘　　要：阿尔茨海默病(AD)是老年期最常见的神经系统退行性疾病,目前临床对其发病机制认识仍较为局限、治疗药物研发滞后。β-淀粉样蛋白(Aβ)级联瀑布假说仍然是目前AD发病机制的主流学说,也是AD疾病修饰药物研发的重要理论基础。近年来,靶向Aβ的免疫治疗药物相继通过美国食品药品监督管理局批准上市或突破性疗法认定,为AD的疾病修饰治疗带来了曙光。文中评述了近年来靶向Aβ疾病修饰治疗临床试验的研究进展,分析总结了以往靶向Aβ疾病修饰治疗临床试验失败的原因。虽然目前靶向Aβ疾病修饰疗法不是十分成熟,但其俨然已成为极具前景的AD药物研发策略。Alzheimer′s disease(AD)is the most common neurodegenerative disease.Its etiology and pathogenesis remain unclear.Since its inception,the amyloid-β(Aβ)cascade hypothesis has dominated the field of AD research and has provided the intellectual framework for disease-modifying therapies.Nowadays,many Aβ-targeted therapies have been accelerated approval or received Food and Drug Administration′s breakthrough therapy designation which offers a new dawn for disease-modifying treating AD.This article reviews the research progress of clinical trials of Aβ-targeting modification therapies,and summarizes the lessons learned from the clinical failure with several classes of anti-Aβdrugs.Although many challenges remain,anti-Aβtherapies have become a promising treatment strategy for AD.

关 键 词：阿尔茨海默病 Β淀粉样蛋白 临床试验 疾病修饰治疗 

分 类 号：R749.16[医药卫生—神经病学与精神病学]