Disease modification and Neuroprotection in neurodegenerative disorders

作     者：Jeffrey Cummings 

作者机构：Cleveland Clinic Lou Ruvo Center for Brain Health888 W Bonneville AveLas VegasNV 89106USA 

出 版 物：《Translational Neurodegeneration》 (转化神经变性病（英文）)

年 卷 期：2017年第6卷第1期

页      面：232-238页

核心收录：

学科分类：1002[医学-临床医学] 100214[医学-肿瘤学] 10[医学] 

基　　金：JC acknowledges funding from the National Institute of General Medical Sciences(Grant:P20GM109025)and support from Keep Memory Alive 

主　　题：Alzheimer’s disease Frontotemporal dementia Progressive supranuclear palsy Corticobasal degeneration Amyotropic lateral sclerosis Multiple system atrophy Disease modification Disease modifying therapy 

摘      要：Background:Disease modifying therapies(DMTs)are urgently needed for neurodegenerative diseases(NDD)such as Alzheimer’s disease(AD)and many other disorders characterized by protein aggregation and *** advances in understanding the neurobiology of NDD,there are no approved ***:Defining disease-modification is critical to drug-development programs.A DMT is an intervention that produces an enduring change in the trajectory of clinical decline of an NDD by impacting the disease processes leading to nerve cell death.A DMT is neuroprotective,and neuroprotection will result in disease *** modification can be demonstrated in clinical trials by a drug-placebo difference in clinical outcomes supported by a drug-placebo difference on biomarkers reflective of the fundamental pathophysiology of the ***,disease modification can be supported by findings on a staggered start or delayed withdrawal clinical trial *** multiple biomarkers is necessary to support a comprehensive view of disease ***:Disease modification is established by demonstrating an enduring change in the clinical trajectory of an NDD based on intervention in the fundamental pathophysiology of the disease leading to nerve cell *** data are collected in clinical *** defining a DMT will assist in NDD drug development programs.