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阿尔茨海默病研究中提高临床前到临床转化的原则和技术年度研讨会会议报告。

Meeting report of the annual workshop on Principles and Techniques for Improving Preclinical to Clinical Translation in Alzheimer's Disease research.

机构信息

The Jackson Laboratory, Bar Harbor, Maine, USA.

Indiana University School of Medicine, Stark Neurosciences Research Institute, Indianapolis, Indiana, USA.

出版信息

Alzheimers Dement. 2023 Nov;19(11):5284-5288. doi: 10.1002/alz.13093. Epub 2023 Apr 18.

DOI:10.1002/alz.13093
PMID:37070936
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10582196/
Abstract

INTRODUCTION

The second annual 5-day workshop on Principles and Techniques for Improving Preclinical to Clinical Translation in Alzheimer's Disease Research was held October 7-11, 2019, at The Jackson Laboratory in Bar Harbor, Maine, USA, and included didactic lectures and hands-on training. Participants represented a broad range of research across the Alzheimer's disease (AD) field, and varied in career stages from trainees and early stage investigators to established faculty, with attendance from the United States, Europe, and Asia.

METHODS

In line with the National Institutes of Health (NIH) initiative on rigor and reproducibility, the workshop aimed to address training gaps in preclinical drug screening by providing participants with the skills and knowledge required to perform pharmacokinetic, pharmacodynamics, and preclinical efficacy experiments.

RESULTS

This innovative and comprehensive workshop provided training in fundamental skill sets for executing in vivo preclinical translational studies.

DISCUSSION

The success of this workship is expected to translate into practical skills that will enable the goals of improving preclinical to clinical translational studies for AD.

HIGHLIGHTS

Nearly all preclinical studies in animal models have failed to translate to successful efficacious medicines for Alzheimer's disease (AD) patients. While a wide variety of potential causes of these failures have been proposed,deficiencies in knowledge and best practices for translational research are not being sufficiently addressed by common training practices. Here we present proceedings from an annual NIA-sponsored workshop focused specifically on preclinical testing paradigms for AD translational research in animal models aimed at enabling improved preclinical to clinical translation for AD.

摘要

简介

第二届年度为期五天的“改善阿尔茨海默病研究临床前到临床转化原则和技术”研讨会于 2019 年 10 月 7 日至 11 日在美国缅因州巴港的杰克逊实验室举行,包括理论讲座和实践培训。参与者代表了阿尔茨海默病(AD)领域广泛的研究,其职业阶段从受训者和早期研究人员到资深教员各不相同,来自美国、欧洲和亚洲的人员参加了会议。

方法

为了应对临床前药物筛选方面的培训差距,本次研讨会与美国国立卫生研究院(NIH)的严谨性和可重复性倡议保持一致,旨在为参与者提供进行药代动力学、药效学和临床前疗效实验所需的技能和知识。

结果

这次创新性和全面的研讨会为执行体内临床前转化研究提供了基础技能培训。

讨论

预计该研讨会的成功将转化为实用技能,从而提高 AD 的临床前转化研究目标。

亮点

几乎所有在动物模型中的临床前研究都未能转化为治疗阿尔茨海默病(AD)患者的有效药物。尽管已经提出了导致这些失败的各种潜在原因,但在知识和转化研究最佳实践方面的不足并没有通过常见的培训实践得到充分解决。在这里,我们介绍了一个由 NIA 主办的年度研讨会的会议记录,该研讨会专门针对 AD 动物模型的临床前测试范式,旨在提高 AD 的临床前到临床转化。