Antiepileptogenesis and disease modification: Progress, challenges, and the path forward-Report of the Preclinical Working Group of the 2018 NINDS-sponsored antiepileptogenesis and disease modification workshop.

Antiepileptogenesis and disease modification: Progress, challenges, and the path forward-Report of the Preclinical Working Group of the 2018 NINDS-sponsored antiepileptogenesis and disease modification workshop.
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DOI:
10.1002/epi4.12490
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发表时间:
2021-06
期刊:
影响因子:
3
通讯作者:
Klein B
Klein B
中科院分区:
医学2区
文献类型:
--
作者:
Galanopoulou AS;Löscher W;Lubbers L;O'Brien TJ;Staley K;Vezzani A;D'Ambrosio R;White HS;Sontheimer H;Wolf JA;Twyman R;Whittemore V;Wilcox KS;Klein B

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癫痫是最常见的慢性脑部疾病之一,通常与认知、行为或其他医疗状况有关。对预防、改善或治愈癫痫及其伴随并发症的疗法的需求是癫痫研究和公共卫生的首要任务。 2018年,国家神经疾病和中风研究所(NINDS)召开了题为“加速抗癫痫发生和疾病缓解疗法的开发”的研讨会,汇集了临床前和临床研究人员以及行业和监管机构的代表,讨论并提出了加速抗癫痫发生(AEG)和疾病缓解(DM)新疗法开发的路线图。本报告总结了临床前科学工作组的讨论和建议。介绍了旨在改善基础设施和转化为临床试验的正在进行的研究举措的 AEG/DM 临床前合作研究项目的进展亮点。与临床研究相比,临床前癫痫研究的机遇和挑战得到了广泛讨论,因为它们涉及跨病因和年龄的特定癫痫类型的建模、AG/DM研究中临床前模型的利用、策略和研究设计,以及与透明度、数据共享和报告研究结果有关的事项。讨论并提出了一系列关于研究举措、基础设施、研讨会、宣传和扩大癫痫研究边界的机会的建议,作为有用的举措,可以帮助制定加速和优化临床前转化 AEG/DM 癫痫研究的路线图。
Epilepsy is one of the most common chronic brain diseases and is often associated with cognitive, behavioral, or other medical conditions. The need for therapies that would prevent, ameliorate, or cure epilepsy and the attendant comorbidities is a priority for both epilepsy research and public health. In 2018, the National Institute of Neurological Disease and Stroke (NINDS) convened a workshop titled “Accelerating the Development of Therapies for Antiepileptogenesis and Disease Modification” that brought together preclinical and clinical investigators and industry and regulatory bodies’ representatives to discuss and propose a roadmap to accelerate the development of antiepileptogenic (AEG) and disease‐modifying (DM) new therapies. This report provides a summary of the discussions and proposals of the Preclinical Science working group. Highlights of the progress of collaborative preclinical research projects on AEG/DM of ongoing research initiatives aiming to improve infrastructure and translation to clinical trials are presented. Opportunities and challenges of preclinical epilepsy research, vis‐à‐vis clinical research, were extensively discussed, as they pertain to modeling of specific epilepsy types across etiologies and ages, the utilization of preclinical models in AG/DM studies, and the strategies and study designs, as well as on matters pertaining to transparency, data sharing, and reporting research findings. A set of suggestions on research initiatives, infrastructure, workshops, advocacy, and opportunities for expanding the borders of epilepsy research were discussed and proposed as useful initiatives that could help create a roadmap to accelerate and optimize preclinical translational AEG/DM epilepsy research.
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