Basic Research, Clinical Trial Infrastructure, and Community Engagement to Support Drug Development for ALS

Meeting Objective: Most cases of ALS, a progressive neurodegenerative disease, have no known cause or cure. Disease pathophysiology is incompletely characterized—approximately 10% of ALS cases are linked to genetic biomarkers, while the other 90% of cases are classified as sporadic—presenting challenges in trial design and patient enrollment. While much progress has been made in basic and clinical ALS research to date, there is still an outstanding need for enhanced evidence generation on the safety and effectiveness of investigational ALS therapeutics across disease stages. In this workshop, participants will discuss challenges impeding therapeutic development for ALS (e.g., issues associated with disease characterization, endpoint development, and the maintenance of a robust research infrastructure) as well as ways to overcome them.