Beghi Ettore, Chiò Adriano, Couratier Philippe, Esteban Jesùs, Hardiman Orla, Logroscino Giancarlo, Millul Andrea, Mitchell Douglas, Preux Pierre-Marie, Pupillo Elisabetta, Stevic Zorica, Swingler Robert, Traynor Bryan J, Van den Berg Leonard H, Veldink Jan H, Zoccolella Stefano
Istituto di Ricerche Farmacologiche "Mario Negri", Via G. la Masa 19, 20156 Milano, Italy.
Amyotroph Lateral Scler. 2011 Jan;12(1):1-10. doi: 10.3109/17482968.2010.502940. Epub 2010 Aug 11.
Abstract Effective treatments for amyotrophic lateral sclerosis (ALS) have remained elusive. Only riluzole, a drug thought to affect glutamate metabolism, improves survival albeit to modest extent. Explanations for the negative results of therapeutic trials include a likely heterogeneity, both in disease susceptibility and pathogenic mechanisms, and faulty methodology of clinical trials. Further understanding of these factors will lead to improvements in patient stratification, and in the design of future clinical trials.
摘要 肌萎缩侧索硬化症（ALS）的有效治疗方法一直难以捉摸。只有利鲁唑，一种被认为会影响谷氨酸代谢的药物，能提高患者生存率，尽管提高幅度不大。治疗试验结果为阴性的原因包括疾病易感性和致病机制可能存在异质性，以及临床试验方法存在缺陷。对这些因素的进一步了解将有助于改善患者分层以及未来临床试验的设计。
