肌萎缩侧索硬化症发病机制及药物研究进展  

Advances of pathogenesis and drug development in amyotrophic lateral sclerosis

作　　者：周昱君 陈秋宇 孙卓 唐婧姝 兰嘉琦[1] 吴镭 彭英[1] ZHOU Yu-jun;CHEN Qiu-yu;SUN Zhuo;TANG Jing-shu;LAN Jia-qi;WU Lei;PENG Ying(Institute of Materia Medica,Chinese Academy of Medical Sciences and Peking Union Medical College,Beijing 100050,China)

机构地区：[1]中国医学科学院北京协和医学院药物研究所,北京100050

出　　处：《中国药理学通报》2024年第2期201-207,共7页Chinese Pharmacological Bulletin

基　　金：国家自然科学基金资助项目(No 82073835);中国医学科学院医学与健康科技创新工程(No 2021-I2M-1-054)。

摘　　要：肌萎缩侧索硬化症(amyotrophic lateral sclerosis,ALS)是一种病因未明的运动神经元疾病,同时累及上、下运动神经元,患者表现出进行性加重的肌肉萎缩、无力、瘫痪,最终死于呼吸衰竭。ALS具有进展快、致死率高的特点,该疾病异质性强,发病机制研究不明确,缺乏有效的治疗药物。因此,探究ALS疾病病理机制、助力新药研发,解决未被满足的临床需求具有重大的科学价值及社会意义。该文针对ALS的发病机制及治疗药物的研究现状进行综述,希望为ALS药物研发提供思路。Amyotrophic lateral sclerosis(ALS)is a fatal neurodegenerative disease affecting both upper and lower motor neurons.ALS patients develop progressive muscle atrophy,muscle weak and paralysis,finally died of respiratory failure.ALS is characterized by fast aggression and high mortality.What′s more,the disease is highly heterogeneous with unclear pathogenesis and lacks effective drugs for therapy.In this review,we summarize the main pathological mechanisms and the current drugs under development for ALS,which may provide a reference for the drug discovery in the future.

关 键 词：肌萎缩侧索硬化症 氧化应激 蛋白聚集体 线粒体障碍 谷氨酸兴奋性毒性 神经炎症 轴突损伤 骨骼肌萎缩 

分 类 号：R746.402[医药卫生—神经病学与精神病学] R746.405.3[医药卫生—临床医学]