Theme 9 Clinical trials and trial design

Abstract

Background: NEALS is a non-profit organization that aims to successfully promote both research and awareness of ALS, encouraging collaborations among clinicians, researchers, and industry through a commitment to open scientific communication. The efforts of NEALS members are recognized by disease foundations, research groups, and industry, generating critical support through awards and project funding.Objectives: Bringing together a group of distinguished scientists and researchers over the nearly quarter century since its inception, NEALS has developed novel research approaches and recruited a number of new investigators into the field of ALS research in an effort to accelerate ALS clinical trials through academic input and partnership.Methods: The NEALS Consortium adheres to an inclusive policy of adding clinical sites wherever there is interest and expertise, improving access to trials in regions where trial participation was not previously available. NEALS sites include 127 academic ALS trial centers in the United States, Canada, Mexico, Lebanon, Italy, Australia and Israel.Education and Participation of the PALS Community in Clinical ResearchAnnual Clinical Research Learning InstitutePALS & CALS as Research AmbassadorsAdvocacy efforts by Research AmbassadorsNEALS website & clinical trial database: http://www.
neals.orgResults: The numerous trials run by NEALS-affiliated Principal Investigators and/or Centers is used as a broad benchmark of success for the Consortium. Additionally, the growth of participation in studies by people with ALS has been used as a similar outcome measure when assessing the success of the organization. The research done by NEALS members, paired with the membership's commitment to a coordinated effort, give the NEALS Consortium a unique role, one with top credibility in the community of people with ALS (PALS). The strong relationship between the Consortium's research members and PALS is substantiated by the increasing participation of PALS in the organization's research efforts. Since 1999, 125,286 PALS have participated in NEALS studies. In 2018 alone, 4,173 people with ALS participated in NEALS trials, up significantly from 1999's participation (n=101).Discussion and conclusions: It is more critical than ever for healthcare professionals to underscore the importance of clinical research into the causes and treatments of ALS and associated conditions. In this vision for the future, by involving more people in ALS research and ensuring that they and those around them are properly informed about the disease, public knowledge regarding rare disease research grows, allowing the research community to give back to the patient population through science breakthroughs and increased disease knowledge.