Workshop Will Update ALS Clinical Trial Guidelines

Patients, their families and caregivers, researchers and clinicians are invited to gain insight into the testing process for experimental therapies to treat amyotrophic lateral sclerosis (ALS) via a live webcast this month. The ALS Clinical Trials Guidelines 2016 Workshop, an international effort that seeks to renew and update the current ALS Clinical Trials Guidelines, will be held March 17-19 at the Arlie Hotel and Conference Center in Warrenton, Va.

The workshop, which is supported in part by MDA, will be webcast live for ALS patients and families, researchers and clinicians who wish to follow the proceedings.

There is only one drug approved for the treatment of ALS at this time. The lack of effective therapies indicates a need for critical reassessment of how drugs are developed and tested for the disease. Clinical trial guidelines based on an evidence-based medicine approach by the international community to direct drug development and clinical trial testing for ALS were published in 1999. Following the rapid pace of discovery in recent years including advances in gene identification and biomarker development for ALS, these guidelines are now out-of-date and in need of revision.

MDA is supporting the effort to gather international leaders in the ALS community including stakeholders from academia, pharma, patient-advocacy organizations, and the federal government to update clinical trial guidelines for ALS. Representatives from MDA will attend and actively participate in the workshop, which will include the review of important topics such as translation of drug candidates to clinical trials, biomarker use, outcome measures, study design, and patient perspectives. The workshop will conclude with the drafting of new guidelines which will be published and disseminated for implementation by the ALS drug development community.

If you are interested in following the live webcast, you may register at

The generation of new guidelines to direct ALS drug development and clinical trial testing will provide a structure for more effective clinical trials that will help accelerate the development of safe and effective treatments for ALS.