North East ALS Study Group

Abstract

The Northeast ALS consortium was formed in 1995 with nine sites interested in collaborative clinical research. We have since expanded to over 30 participating sites across much of the US. We also hope to collaborate with clinical researchers in Europe and Canada for Phase III efficacy trials that require the involvement of many sites. Our mission statement is to cooperate to plan, perform, analyze and report controlled clinical trials in ALS, with the goal of improving treatment for patients with ALS. The concept we had was that a large group could rapidly execute high quality pivotal trials in ALS and provide a mechanism to test more therapies. Critical to the success of the consortium is close collaboration with many basic scientists who study the causes of the disease and who have various models for screening compounds. The group has developed several different approaches to testing therapies depending on the stage of drug development. We have established intra- and inter-rater reliability across the centers for muscle strength and pulmonary measures, and motor unit number estimates. We continue to explore other measurements that might be useful in this disease. The NEALS organization decided from the onset to be very inclusive; anyone wishing to be involved can become a member. The executive committee has two co-chairs, who are elected every six years, and two elected site investigators elected for three-year terms. The scientific advisory committee and statistical consultants also participate on the executive committee. We created a number of working groups including members interested in clinical trial design, biomarkers, and other collaborative research projects.