肌萎缩侧索硬化（amyotrophic lateral sclerosis，ALS）是一种发病后快速进展的致命性神经退行性疾病，目前尚无有效药物能够延缓其疾病进展或改善其临床症状。大量的临床实践表明，中医药治疗可以延缓 ALS 的疾病进展，并显著改善患者的临床症状，但是对其作用机制仍不清楚。近年来的基础研究发现，ALS 的发病和疾病进展均与兴奋性神经元毒性、线粒体功能障碍、轴浆转运障碍、神经营养因子缺陷以及小胶质细胞和星形胶质细胞功能障碍等有关。中医药可能通过干预上述途径，发挥治疗 ALS 的作用。本文提出可以通过已经建立的 ALS 动物模型，研究中医药治疗 ALS 疗效的多靶点分子机制，为中医药治疗 ALS 提供基于分子生物学和分子遗传学的依据。

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease with rapid progression after onset. At present, there are no effective drugs to delay the disease progression or improve its clinical symptoms. A large number of clinical practices have shown that intervention with traditional Chinese medicine can delay the progression of ALS and significantly improve the clinical symptoms of patients, but its mechanism is still unclear. In recent years, basic studies have found that the pathogenesis and progression of ALS are related to excitatory neurotoxicity, mitochondrial dysfunction, axoplasmic transport dysfunction, neurotrophic factor deficiency, microglia and astrocyte dysfunction. Traditional Chinese medicine may play a role in the treatment of ALS by intervening in the above ways. This paper points out that the established animal model of ALS can be used to study the multi-target molecular mechanism of traditional Chinese medicine in the treatment of ALS, and provide the basis of molecular biology and genetics for traditional Chinese medicine in the treatment of ALS.