Translating preclinical insights into effective human trials in ALS

被引:31
作者
DiBernardo, Allitia B. [1 ]
Cudkowicz, Merit E. [1 ]
机构
[1] Massachusetts Gen Hosp, Neurol Clin Trials Unit, Dept Neurol, Charlestown, MA 02129 USA
来源
BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE | 2006年 / 1762卷 / 11-12期
关键词
amyotrophic lateral sclerosis; clinical trial; preclinical model; drug evaluation;
D O I
10.1016/j.bbadis.2006.03.007
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
Amyotrophic lateral sclerosis (ALS) is a rapidly progressive, adult-onset neurodegenerative disease characterized by selective dysfunction and death of motor neurons in the brain and spinal cord. The disease is typically fatal within 3-5 years of symptom onset. There is no known cure and only riluzole, which was approved by the FDA in 1996 for treatment of ALS, has shown some efficacy in humans. Preclinical insights from model systems continue to furnish ample therapeutic targets, however, translation into effective therapies for humans remains challenging. We present an overview of clinical trial methodology for ALS, including a summary rationale for target selection and challenges to ALS clinical research. (c) 2006 Elsevier B.V. All rights reserved.
引用
收藏
页码:1139 / 1149
页数:11
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