Access for All in ALS (ALL ALS) West Clinical Coordinating Center

肌萎缩侧索硬化症(ALS)是一种进展迅速、最终致命的神经退行性疾病，在美国约有32,000例。据估计，终身患病的风险为1/400，平均每年美国新诊断的病例为5000例。鉴于在将实验室结果转化为临床有效疗法方面的许多失败，从不同的肌萎缩侧索硬化症队列中产生高质量、临床注释的纵向患者衍生生物样本以及由此产生的下游分析预计将加速有效新药的开发，以延长肌萎缩侧索硬化症患者的生命。可以利用公私合作伙伴关系(PPP)来迅速推进管理科学和科学研究，以支持和加速有效新药的开发，以延长肌萎缩侧索硬化症患者的生命。 为了实现新成立的ALS公私合作伙伴关系(PPP)ACT的研究目标，以加速药物开发，我们提议建立一个新的全国性ALS临床研究联盟-ALL ALS(ALL ALS)联盟。 所有ALS将有2个协调中心(ALL ALS东部协调中心和ALL ALS西部协调中心)，这两个中心将共同管理和监督美国和波多黎各的34个临床地点。ALL ALS WEST协调中心将设在圣约瑟夫医院和医疗中心的巴罗神经研究所。在我们的目标肌萎缩侧索硬化症自然病史研究和联合体不断取得成功的基础上，ALL西部协调中心将继续管理目前对目标肌萎缩侧索硬化症自然史研究做出贡献的临床站点，以及另外10个新的临床站点。 新的ALL ALS联盟将提供大规模、集中化和易于访问的基础设施，用于收集和存储广泛的数据：其中包括从1)ALS患者、2)有ALS风险的个人和3)健康对照收集的纵向临床测量和生物液。为了促进广泛的人口代表性，将从远程和面对面的访问中收集数据，许多诊所将位于少数民族代表不足的地理区域。此外，为便利今后的研究和协作，将在一个单一的中央数据门户中收集和统一数据。

Amyotrophic lateral sclerosis (ALS) is a rapidly progressive, ultimately fatal, neurodegenerative disease with approximately 32,000 cases in the United States (US). With an estimated lifetime risk of 1/400, an average of 5,000 new US cases are diagnosed every year. Given the numerous failures in translating laboratory results into clinically effective therapies, the generation of high quality, clinically annotated longitudinal patient-derived biosamples from diverse ALS cohorts and resulting downstream analyses are expected to accelerate the development of effective, new drugs to extend life for people living with ALS A public private partnership (PPP) can be leveraged to rapidly advance regulatory science and scientific research to support and accelerate development of effective new drugs to extend life for people living with ALS. To operationalize the research objectives of the newly formed ACT for ALS Public Private Partnership (PPP) to accelerate drug development, we propose creation of a new national ALS clinical research consortium, the Access for All in ALS (ALL ALS) Consortium. ALL ALS will have 2 coordinating centers (the ALL ALS East Coordinating Center and the ALL ALS West Coordinating Center) which will together administer and oversee 34 clinical sites across the United States and Puerto Rico. The ALL ALS West Coordinating Center will be based at the Barrow Neurological Institute at St Joseph’s Hospital and Medical Center. Building upon the ongoing success of our Target ALS natural history study and consortium, the ALL ALS West Coordinating Center will continue to administer clinical sites currently contributing to the Target ALS natural history study, along with 10 additional new clinical sites. The new ALL ALS Consortium will provide large-scale, centralized, and readily accessible infrastructure for collection and storage of a wide range of data: these include longitudinally collected clinical measures and biofluids from 1) people living with ALS, 2) individuals at risk for developing ALS, and 3) healthy controls. To foster wide representation across populations, data will be collected from both remote and in person visits, and many clinical sites will be located in geographical areas with under-represented minorities. Furthermore, to facilitate future research and collaboration, data will be collected and harmonized in a single central data portal.