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应对在开发改变痴呆症治疗方法方面存在的差距。

Tackling gaps in developing life-changing treatments for dementia.

作者信息

Mauricio Rui, Benn Caroline, Davis John, Dawson Gerry, Dawson Lee A, Evans Alison, Fox Nick, Gallacher John, Hutton Mike, Isaac John, Jones Declan N C, Jones Lesley, Lalli Giovanna, Libri Vincenzo, Lovestone Simon, Moody Catherine, Noble Wendy, Perry Hugh, Pickett James, Reynolds David, Ritchie Craig, Rohrer Jonathan D, Routledge Carol, Rowe James, Snyder Heather, Spires-Jones Tara, Swartz Jina, Truyen Luc, Whiting Paul

机构信息

Alzheimer's Research UK, Cambridge, UK.

Dementia Discovery Fund, London, UK.

出版信息

Alzheimers Dement (N Y). 2019 Jun 24;5:241-253. doi: 10.1016/j.trci.2019.05.001. eCollection 2019.

Abstract

Since the G8 dementia summit in 2013, a number of initiatives have been established with the aim of facilitating the discovery of a disease-modifying treatment for dementia by 2025. This report is a summary of the findings and recommendations of a meeting titled "Tackling gaps in developing life-changing treatments for dementia", hosted by Alzheimer's Research UK in May 2018. The aim of the meeting was to identify, review, and highlight the areas in dementia research that are not currently being addressed by existing initiatives. It reflects the views of leading experts in the field of neurodegeneration research challenged with developing a strategic action plan to address these gaps and make recommendations on how to achieve the G8 dementia summit goals. The plan calls for significant advances in (1) translating newly identified genetic risk factors into a better understanding of the impacted biological processes; (2) enhanced understanding of selective neuronal resilience to inform novel drug targets; (3) facilitating robust and reproducible drug-target validation; (4) appropriate and evidence-based selection of appropriate subjects for proof-of-concept clinical trials; (5) improving approaches to assess drug-target engagement in humans; and (6) innovative approaches in conducting clinical trials if we are able to detect disease 10-15 years earlier than we currently do today.

摘要

自2013年八国集团痴呆症峰会以来,已发起多项倡议,旨在推动在2025年前发现可改变疾病进程的痴呆症治疗方法。本报告总结了2018年5月由英国阿尔茨海默病研究组织主办的题为“解决痴呆症改变生活疗法开发中的差距”会议的研究结果和建议。该会议的目的是确定、审查和突出痴呆症研究中现有倡议目前未涉及的领域。它反映了神经退行性疾病研究领域顶尖专家的观点,这些专家面临着制定战略行动计划以填补这些差距的挑战,并就如何实现八国集团痴呆症峰会目标提出建议。该计划要求在以下方面取得重大进展:(1)将新发现的遗传风险因素转化为对受影响生物过程的更好理解;(2)增强对选择性神经元复原力的理解,为新的药物靶点提供信息;(3)促进可靠且可重复的药物靶点验证;(4)为概念验证临床试验合理且基于证据地选择合适的受试者;(5)改进评估人体药物靶点结合情况的方法;以及(6)如果我们能够比目前提前10 - 15年检测到疾病,在开展临床试验方面采用创新方法。

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