What can we learn from the edaravone development program for ALS?

Research output: Contribution to journalReview articlepeer-review

Abstract

Edaravone’s development into an ALS therapeutic has been a process which began with preclinical studies regarding its potential in targeting ALS. Despite edaravone’s inability to show benefit in a general ALS population, an important post-hoc analysis showed that a clinical subset of patients had benefit. Most importantly, a subsequent study examining the capacity of edaravone to have benefit in this specific subset of ALS patients was successful in meeting its primary outcome measures. Questions regarding whether the dosing regimen could be simplified or improved, the duration of the effects, and the timing of the potential treatment to different stages of disease remain to be answered. However, the benefit of this compound in delivering a meaningful therapy to ALS patients and the lessons learned with regard to its development should widen interest in clinical research so that additional strategies for treating ALS may become available to patients.

Original languageEnglish (US)
Pages (from-to)98-103
Number of pages6
JournalAmyotrophic Lateral Sclerosis and Frontotemporal Degeneration
Volume18
DOIs
StatePublished - Oct 31 2017

Keywords

  • Therapy
  • clinical trial
  • drug
  • motor neuron disease

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

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