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

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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.