The United States National Amyotrophic Lateral Sclerosis (ALS) Registry Advances Research Domestically and Internationally


Translational Research

Poster Number: 297


Paul Mehta, MD, National ALS Registry - CDC/ATSDR, Jaime Raymond MPH, MPH, National ALS Registry

OBJECTIVE: Describe how the National ALS Registry supports and advances research in the United States and abroad.

BACKGROUND: The National Amyotrophic Lateral Sclerosis Registry, maintained by the Centers for Disease Control and Prevention/Agency for Toxic Substances and Disease Registry (CDC/ATSDR), is a multi-faceted research platform. One of the purposes of the Registry, as defined by Congress, “is to facilitate research.” In addition to registering patients with ALS and collecting epidemiological data, a National ALS Biorepository was added in 2015 after the completion of a pilot project. The purpose of the Biorepository is to expand ALS research in areas such as genetics, biomarker identification, environmental exposures, and disease progression. Because recruiting persons with ALS can be difficult and time-consuming, the Registry can assist with recruiting persons with ALS into clinical trials and research studies. The Registry can also provide de-identified self-reported epidemiological data and/or biological samples for a subset of persons in the Registry.

DESIGN/METHODS: CDC/ATSDR developed mechanisms that allow eligible persons with ALS to be informed about clinical trials and research studies, provide specimens to the Biorepository, and self-report epidemiologic data securely. Researchers, both domestic and international, can apply to receive data from the Registry and samples from the Biorepository as well as have recruitment emails sent for their studies. The Registry also provides funding for investigator-initiated R01 grants.

RESULTS: To date, over 65 institutions (pharmaceutical companies and academia) have used the Registry to recruit for their clinical trials and studies. These include notable clinical trials for Amylyx Pharmaceuticals, Brainstorm Cell Therapeutics, Biogen, Inc., Cytokinetics, Inc., and others. Over 60,000 specimens (e.g., blood, hair) have been collected nationally from >1,500 persons with ALS and >180 post-mortem autopsies (e.g., tissue, bone) have been conducted for the National ALS Biorepository and are available for dissemination to researchers. Survey data are available for researchers from 18 risk factor modules. Over 100,000 surveys have been completed, and the Registry has funded 23 research grants to date.

CONCLUSIONS: The National ALS Registry is a multi-faceted research platform that provides researchers access to a large national group of ALS and data for their research.