Berry James D, Cudkowicz Merit E
Massachusetts General Hospital, Department of Neurology, Neurology Clinical Trials Unit, 149 Thirteenth Street, Suite 2274, Charlestown, MA 02129, USA.
Clin Investig (Lond). 2011 Oct;1(10):1375-1389. doi: 10.4155/cli.11.127.
Amyotrophic lateral sclerosis is a devastating neurodegenerative disease caused by loss of motor neurons. Its pathophysiology remains unknown, but progress has been made in understanding its genetic and biochemical basis. Clinical trialists are working to translate basic science successes into human trials with more efficiency, in the hope of finding successful treatments. In the future, new preclinical models, including patient-derived stem cells may augment transgenic animal models as preclinical tools. Biomarker discovery projects aim to identify markers of disease onset and progression for use in clinical trials. New trial designs are reducing study time, improving efficiency and helping to keep pace with the increasing rate of basic and translational discoveries. Ongoing trials with novel designs are paving the way for amyotrophic lateral sclerosis clinical research.
肌萎缩侧索硬化症是一种由运动神经元丧失引起的毁灭性神经退行性疾病。其病理生理学仍然未知，但在理解其遗传和生化基础方面已取得进展。临床试验人员正致力于更高效地将基础科学成果转化为人体试验，以期找到成功的治疗方法。未来，包括患者来源干细胞在内的新的临床前模型可能会作为临床前工具补充转基因动物模型。生物标志物发现项目旨在识别疾病发作和进展的标志物，用于临床试验。新的试验设计正在缩短研究时间、提高效率，并有助于跟上基础和转化研究发现的增长速度。正在进行的采用新颖设计的试验为肌萎缩侧索硬化症的临床研究铺平了道路。
