ALS Clinical Trials Guidelines

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 DESCRIPTION (provided by applicant): Amyotrophic lateral sclerosis (ALS) is a devastating disease that causes relentlessly progressive skeletal muscle paralysis and eventual respiratory failure; it invariably leads to the death of most patients within 3 to 5 years. The fact that ~50 clinical trials have been performed in the past half-century and riluzole, with modest benefits, is the only ALS medication we can prescribe indicates a serious need for critical reassessment of the methods employed in ALS drug development at every level. The necessity for effective medications is urgent -- patients with ALS and those who care for them are desperate for new treatment. The meeting to create the previous ALS clinical trial guidelines was held in 1998, and these guidelines, based on expert consensus, were published 15 years ago in 1999, rendering them completely outdated in light of advances in clinical trials in ALS, emerging biomarkers, and overall trial methodology. Accordingly, we propose to hold a major international conference to review current issues in clinical trials in ALS and formulate new and updated ALS clinical trial guidelines using an evidence-based medicine (EBM) approach. We specifically aim: 1. To finalize the formation of an Organizing Committee and Advisory Board of international ALS physician and physician-scientist volunteers to develop and organize an international meeting that will update the present ALS clinical trial guidelines; 2. To hold an international conference o review the issues and current status of clinical trials; 3. To discuss and develop guidelines for ALS RCTs; and 4.To publish the guidelines in major neurology journals and promote implementation of these guidelines into future ALS trials. While we propose this U13 grant to support young clinical trialists and biostatisticians, basic scientists, and pharmacologists in ALS with the help of additional funding we plan to have representation and participation from all ALS stakeholders, including officers of regulatory and funding agencies, clinical trialists, advocacy groups, and patients and their caregivers. A web-conference will be also available for those who cannot attend the meeting in person. We believe that the new trial guidelines, when widely disseminated and actively implemented, will result in more effective and efficient trials, leading to the development of better medications in the future in ALS.