Hampel Harald, Schneider Lon S, Giacobini Ezio, Kivipelto Miia, Sindi Shireen, Dubois Bruno, Broich Karl, Nisticò Robert, Aisen Paul S, Lista Simone
AXA Research Fund and UPMC Chair, Paris, France.
Expert Rev Neurother. 2015 Jan;15(1):83-105. doi: 10.1586/14737175.2015.995637. Epub 2014 Dec 24.
Worldwide multidisciplinary translational research has led to a growing knowledge of the genetics and molecular pathogenesis of Alzheimer's disease (AD) indicating that pathophysiological brain alterations occur decades before clinical signs and symptoms of cognitive decline can be diagnosed. Consequently, therapeutic concepts and targets have been increasingly focused on early-stage illness before the onset of dementia; and distinct classes of compounds are now being tested in clinical trials. At present, there is a growing consensus that therapeutic progress in AD delaying disease progression would significantly decrease the expanding global burden. The evolving hypothesis- and evidence-based generation of new diagnostic research criteria for early-stage AD has positively impacted the development of clinical trial designs and the characterization of earlier and more specific target populations for trials in prodromal as well as in pre- and asymptomatic at-risk stages of AD.
全球多学科转化研究使人们对阿尔茨海默病（AD）的遗传学和分子发病机制有了越来越多的了解，这表明病理生理大脑改变早在认知能力下降的临床体征和症状能够被诊断出来的几十年前就已发生。因此，治疗理念和靶点越来越多地聚焦于痴呆症发作前的早期疾病阶段；目前不同种类的化合物正在临床试验中接受测试。目前，越来越多的人达成共识，即AD在延缓疾病进展方面的治疗进展将显著减轻不断扩大的全球负担。基于新的假设和证据生成的早期AD诊断研究标准，对临床试验设计的发展以及前驱期以及AD的前期和无症状风险期试验中更早期、更特定目标人群的特征描述产生了积极影响。
