Vincent Andrea M, Sakowski Stacey A, Schuyler Adam, Feldman Eva L
Department of Neurology, University of Michigan, Ann Arbor 48109, United States.
Drug Discov Today. 2008 Jan;13(1-2):67-72. doi: 10.1016/j.drudis.2007.10.011. Epub 2007 Nov 26.
Significant progress in understanding the cellular mechanisms of motor neuron degeneration in amyotrophic lateral sclerosis (ALS) has not been matched with the development of therapeutic strategies to prevent disease progression. The multiple potential causes and relative rarity of the disease are two significant factors that make drug development and assessment in clinical trials extremely difficult. We review recent progress in promoting therapeutics into clinical trials and highlight the value of moderate throughput screening for the acceleration and improvement of drug design.
在理解肌萎缩侧索硬化症（ALS）中运动神经元变性的细胞机制方面取得了重大进展，但在开发预防疾病进展的治疗策略方面却没有相应进展。该疾病的多种潜在病因以及相对罕见性是使药物开发和临床试验评估极为困难的两个重要因素。我们回顾了将治疗方法推进到临床试验的最新进展，并强调了适度高通量筛选对于加速和改进药物设计的价值。
