Aggarwal Swati, Cudkowicz Merit
Department of Neurology, Massachusetts General Hospital, Neurology Clinical Trials Unit, Charlestown, Massachusetts 02129, USA.
Neurotherapeutics. 2008 Oct;5(4):516-27. doi: 10.1016/j.nurt.2008.08.002.
Tremendous advances in our understanding of pathogenesis of amyotrophic lateral sclerosis (ALS) have provided a rich pipeline of drugs for clinical trialists. At least 32 unique compounds have been tested. Nevertheless, riluzole is currently the only treatment that prolongs survival. We present a critical overview of past clinical trials, how therapies are selected for testing in people, challenges with ALS clinical trial design and conduct, and ways to best move forward.
我们对肌萎缩侧索硬化症（ALS）发病机制的理解取得了巨大进展，为临床试验人员提供了丰富的药物研发渠道。至少32种独特的化合物已进行了测试。然而，利鲁唑是目前唯一能延长生存期的治疗方法。我们对过去的临床试验进行了批判性概述，介绍了如何选择用于人体测试的疗法、ALS临床试验设计与实施面临的挑战，以及未来最佳的推进方向。
