肌萎缩侧索硬化的新药研发与临床试验动态  

New drug development and clinical trial progress of amyotrophic lateral sclerosis

作　　者：赵丽 邱彦 金辉[1,2] 万健[1] ZHAO Li;QIU Yan;JIN Hui;WAN Jian(Clinical Trial Unit,Shanghai Pudong New Area People’s Hospital,SHANGHAI 201299,China;Pharmacy Department,Shanghai Pudong New Area People’s Hospital,SHANGHAI 201299,China)

机构地区：[1]上海市浦东新区人民医院临床试验机构,上海201299 [2]上海市浦东新区人民医院药剂科,上海201299

出　　处：《中国新药与临床杂志》2025年第3期235-240,共6页Chinese Journal of New Drugs and Clinical Remedies

基　　金：上海市浦东新区卫生健康委员会重要薄弱学科建设(PWZbr2022-13)。

摘　　要：肌萎缩侧索硬化(ALS)是一种致命的神经退行性病变,有迫切的、未被满足的治疗需求,目前有多个治疗ALS的新靶点药物在研发中。磷酸肌醇激酶PIKfyve在ALS发病和进展中发挥关键作用,抑制PIKfyve对ALS有明确的治疗效果。抑制PIKfyve的化学小分子药物AIT-101在临床试验中显示出治疗ALS的良好药效和安全性,且可口服,但体内药效时间短暂;反义寡核苷酸药物AS-202在体内抑制PIKfyve的时间更长,即将推进到临床试验中用于治疗ALS和其他神经退行性疾病。Amyotrophic lateral sclerosis(ALS)is a fatal neurodegenerative disease with urgent unmet medical needs.Currently,multiple new drugs with different targets are being developed for ALS therapy.Phosphoinositide kinase PIKfyve plays a crucial role in the pathogenesis and progression of ALS,and new drugs inhibiting PIKfyve exhibit significant therapeutic effects on ALS.The chemical small molecule PIKfyve inhibitor AIT-101 demonstrates excellent efficacy and safety treating ALS in a clinical trial.It can be taken orally,however,with short efficacy time in vivo.AS-202,the antisense oligonucleotide inhibiting PIKfyve,has a much longer efficacy time in vivo,and will soon be developed to treat ALS and other neurodegenerative diseases.

关 键 词：肌萎缩侧索硬化 神经退行性病变 PIKFYVE AI制药 

分 类 号：R971[医药卫生—药品]