Saul R. Korey Department of Neurology, Dominick P. Purpura Department of Neuroscience, Isabelle Rapin Division of Child Neurology, Albert Einstein College of Medicine, Bronx, NY, USA.
Department of Pharmacology, Toxicology, and Pharmacy, University of Veterinary Medicine Hannover, Hannover, Germany.
Epilepsia Open. 2021 Jun;6(2):276-296. doi: 10.1002/epi4.12490. Epub 2021 May 6.
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.
癫痫是最常见的慢性脑部疾病之一,常伴有认知、行为或其他医学状况。无论是癫痫研究还是公共卫生领域,都急需开发能够预防、改善或治愈癫痫及其相关合并症的疗法。2018 年,美国国立神经病学与卒中研究所(NINDS)召开了一次题为“加速抗癫痫发生和疾病修饰疗法开发”的研讨会,召集了临床前和临床研究人员以及工业界和监管机构的代表,共同讨论并提出了加速抗癫痫发生(AEG)和疾病修饰(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。重点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计划中关于 AEG/DM 的合作临床前研究项目的进展。还广泛讨论了临床前癫痫研究与临床研究之间的机遇和挑战,涉及特定病因和年龄的癫痫类型的建模、在 AEG/DM 研究中使用临床前模型,以及策略和研究设计,以及与透明度、数据共享和报告研究结果相关的事项。还讨论并提出了一系列关于研究计划、基础设施、研讨会、宣传和扩大癫痫研究边界的机会的建议,这些建议被认为是有助于制定加速和优化抗癫痫发生和疾病修饰的临床前转化研究路线图的有用举措。