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Alzheimer's disease research and development: a call for a new research road map

机译:阿尔茨海默氏病研究与开发:呼吁制定新的研究路线图

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摘要

Epidemiological projections of the prevalence of Alzheimer's disease (AD) and related dementias, the rapidly expanding population over the age of 65, and the enormous societal consequence on health, economics, and community foretell of a looming global public health crisis. Currently available treatments for AD are symptomatic, with modest effect sizes and limited impact on longer term disease outcomes. There have been no newly approved pharmaceutical treatments in the last decade, despite enormous efforts to develop disease-modifying treatments directed at Alzheimer's-associated pathology. An unprecedented collaborative effort of government, regulators, industry, academia, and the community at-large is needed to address this crisis and to develop an actionable plan for rapid progress toward successfully developing effective treatments. Here, we map out a course of action in four key priority areas, including (1) addressing the fundamental mechanisms of disease, with the goal of developing a core set of research tools, a framework for data sharing, and creation of accessible validated and replicated disease models; (2) developing translational research that emphasizes rapid progress in disease model development and better translation from preclinical to clinical stages, deploying leading technologies to more accurately develop predictive models; (3) preventing AD through the development of robust methods and resources to advance trials and creating fundamental resources such as continuous adaptive trials, registries, data repositories, and instrument development; and (4) innovating public/private partnerships and global collaborations, with mechanisms to incentivize collaborations and investments, develop larger precompetitive spaces, and more rapid data sharing.
机译:流行病学预测:阿尔茨海默氏病(AD)和相关痴呆的患病率; 65岁以上人口的快速增长;以及健康,经济的巨大社会后果以及迫在眉睫的全球性公共卫生危机对社区的预言。当前可用的AD疗法是对症的,效果大小适中且对长期疾病结果的影响有限。尽管在开发针对阿尔茨海默氏病相关疾病的疾病缓解疗法方面做出了巨大努力,但在过去的十年中,还没有新批准的药物疗法。需要政府,监管机构,行业,学术界和广大社区的前所未有的合作努力,以解决这一危机并制定可行的计划,以迅速取得成功,从而成功开发出有效的治疗方法。在这里,我们列出了四个关键优先领域的行动方案,其中包括(1)解决疾病的基本机制,目的是开发一套核心研究工具,一个数据共享框架以及创建可访问的经过验证的和复制疾病模型; (2)开展强调疾病模型开发的快速进展以及从临床前到临床阶段更好的翻译的转化研究,部署领先技术以更准确地开发预测模型; (3)通过开发强大的方法和资源来推进试验并创建基础资源(例如连续的适应性试验,注册,数据存储库和仪器开发)来预防AD; (4)创新公共/私人合作伙伴关系和全球合作,通过激励合作和投资,开发更大的竞争前空间和更快速的数据共享的机制。

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