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Opportunities for Improving Therapy Development in ALS: Roundtable Discussion Summary

January 29, 2014

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:

  • Tremendous progress in understanding new gene discoveries, combined with an acceleration of model development, is likely to generate new disease hypotheses and new potential therapies at a more rapid rate than in the past.
  • Innovations in trial design and infrastructure have helped accelerate the start of clinical trials. These important efforts should continue.
  • Current ALS clinical trials are hindered by a lack of disease progression biomarkers, which would allow a true effect on disease progression to be more rapidly distinguished. Progress in developing such biomarkers is encouraging, with new candidates emerging.
  • Early phase trials are hindered by lack of biomarkers that are linked to the proposed therapeutic target (pharmacodynamic markers). Biomarkers related to therapeutic target would greatly facilitate interpretation of early study trials and allow better selection of therapies to bring forward. Having these biomarkers would inform whether the therapy affected the target and the relevance of the target to disease progression. 
  • ALS patient advocates should be involved in shaping outcome measures for trials, to make clinical measures more meaningful to patients.

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

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