Merck & Co., Inc., Kenilworth, New Jersey, USA.
Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA.
Clin Pharmacol Ther. 2022 Apr;111(4):835-839. doi: 10.1002/cpt.2544. Epub 2022 Mar 2.
Use of early predictive biomarkers of neurodegenerative disease in phase I clinical trials may improve the translation of novel drug therapies from preclinical development through late-stage studies. This article provides a categorical summary of promising biomarker approaches or clinical end points in molecular, cellular, metabolic, electrophysiological, or clinical function that can be used to predict or quantify the progression of neurodegenerative disorders and guide program support.
在 I 期临床试验中使用神经退行性疾病的早期预测性生物标志物,可能会改善从临床前开发到后期研究阶段新型药物疗法的转化。本文对有前途的生物标志物方法或分子、细胞、代谢、电生理或临床功能方面的临床终点进行了分类总结,这些方法或终点可用于预测或量化神经退行性疾病的进展,并指导项目支持。