Sean M. Healey & AMG Center for ALS, Massachusetts General Hospital, Boston, Massachusetts, USA.
Department of Neurology, Columbia University College of Physicians and Surgeons, New York City, New York, USA.
Muscle Nerve. 2024 Dec;70(6):1140-1150. doi: 10.1002/mus.28244. Epub 2024 Sep 18.
Recent progress in therapeutics for amyotrophic lateral sclerosis (ALS) has spurred development and imbued the field of ALS with hope for more breakthroughs, yet substantial scientific gaps persist. This unmet need remains a stark reminder that innovative paradigms are needed to invigorate ALS research. To move toward more informative, targeted, and personalized drug development, the National Institutes of Health (NIH) established a national ALS clinical research consortium called Access for ALL in ALS (ALL ALS). This new consortium is a multi-institutional effort that aims to organize the ALS clinical research landscape in the United States. ALL ALS is operating in partnership with several stakeholders to operationalize the recommendations of the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS) Public Private Partnership. ALL ALS will provide a large-scale, centralized, and readily accessible infrastructure for the collection and storage of a wide range of data from people living with ALS (symptomatic cohort) or who may be at risk of developing ALS (asymptomatic ALS gene carriers). Importantly, ALL ALS is designed to encourage community engagement, equity, and inclusion. The consortium is prioritizing the enrollment of geographically, ethnoculturally, and socioeconomically diverse participants. Collected data include longitudinal clinical data and biofluids, genomic, and digital biomarkers that will be harmonized and linked to the central Accelerating Medicines Partnership for ALS (AMP ALS) portal for sharing with the research community. The aim of ALL ALS is to deliver a comprehensive, inclusive, open-science dataset to help researchers answer important scientific questions of clinical relevance in ALS.
肌萎缩侧索硬化症 (ALS) 的治疗最近取得了进展,这激发了更多的研究和希望,但仍存在大量尚未解决的科学问题。这一未满足的需求提醒我们,需要创新的范式来推动 ALS 研究。为了推进更具信息性、针对性和个性化的药物开发,美国国立卫生研究院 (NIH) 成立了一个名为 ALL ALS 的国家 ALS 临床研究联盟,以实现所有 ALS 患者的治疗机会。这个新的联盟是一个多机构的努力,旨在组织美国的 ALS 临床研究格局。ALL ALS 正在与多个利益相关者合作,以实施加速 ALS 关键疗法获取法案 (ACT for ALS) 公私合作伙伴关系的建议。ALL ALS 将提供一个大规模、集中化和易于访问的基础设施,用于收集和存储来自患有 ALS(症状性队列)或可能有患 ALS 风险的人的广泛数据(无症状 ALS 基因携带者)。重要的是,ALL ALS 旨在鼓励社区参与、公平和包容。该联盟优先招募地理位置、民族文化和社会经济多样化的参与者。收集的数据包括纵向临床数据和生物流体、基因组和数字生物标志物,这些数据将被协调并链接到中央 ALS 加速药物伙伴关系 (AMP ALS) 门户,以便与研究界共享。ALL ALS 的目标是提供一个全面、包容、开放科学的数据集,帮助研究人员回答 ALS 中具有临床相关性的重要科学问题。