Opportunities for improving therapy development in ALS

Abstract

In May 2013, The ALS Association and The Northeast ALS Consortium (NEALS) convened a meeting of stakeholders for a round-table discussion of ways to improve therapy development in ALS. The following overview summarizes issues raised and potential new directions discussed at the meeting. We recommend that future phase II 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, may also improve trials.

Key words::

• Amyotrophic lateral sclerosis
• clinical trials
• biomarkers
• therapy

Acknowledgement

The authors wish to thank Richard Robinson for expert editorial assistance in preparation of this manuscript.

Declaration of interest: Merit Cudkowicz has consulted for GlaxoSmithKline, Cytokinetics and Shire Pharmaceuticals. Lucie Bruijn has consulted for Bristol Myers Squibb, Cytokinetics and Pfizer.