In May 2013, The ALS Association and the Northeast ALS Consortium (NEALS) convened a meeting of stakeholders for a roundtable discussion of ways to improve therapy development in ALS. Scientists and physicians from academic hospitals and industry were joined by patient advocates, representatives from The Food and Drug Administration (FDA), The National Institute of Neurological Disorders and Stroke (NINDS), and institutional review boards. An article presenting an overview of issues raised, and recommendations arising from that discussion, has now been published in the journal Amyotrophic Lateral Sclerosis and Frontotemporal Dementia.
Key points in the article include:
The authors recommend that future early phase clinical trials in ALS proceed when the proposed treatment is directed at targets that are likely to be involved in ALS pathogenesis in a defined subgroup of patients and be accompanied by one or more biomarkers to track both clinical progression and pharmacodynamic engagement of the target. Innovations in trial structure and design, and greater involvement of patient advocates, will impact the efficiency of therapy development in ALS.
Opportunities for Improving Therapy Development in ALS
Lucie Bruijn, Ph.D., Chief Scientist, The ALS Association; Merit Cudkowicz, M.D., M.Sc., Chief of Neurology, Massachusetts General Hospital; and the ALS Clinical Trials Working Group