Drugs in clinical development for the treatment of amyotrophic lateral sclerosis

Expert Opin Investig Drugs. 2017 Apr;26(4):403-414. doi: 10.1080/13543784.2017.1302426. Epub 2017 Mar 14.


Amyotrophic Lateral Sclerosis (ALS) is a fatal motor neuron progressive disorder for which no treatment exists to date. However, there are other investigational drugs and therapies currently under clinical development may offer hope in the near future. Areas covered: We have reviewed all the ALS ongoing clinical trials (until November 2016) and collected in Clinicaltrials.gov or EudraCT. We have described them in a comprehensive way and have grouped them in the following sections: biomarkers, biological therapies, cell therapy, drug repurposing and new drugs. Expert opinion: Despite multiple obstacles that explain the absence of effective drugs for the treatment of ALS, joint efforts among patient's associations, public and private sectors have fueled innovative research in this field, resulting in several compounds that are in the late stages of clinical trials. Drug repositioning is also playing an important role, having achieved the approval of some orphan drug applications, in late phases of clinical development. Endaravone has been recently approved in Japan and is pending in USA.

Keywords: ALS; biomarkers; cell therapy; clinical trials; new drugs.

Publication types

  • Review

MeSH terms

  • Amyotrophic Lateral Sclerosis / drug therapy*
  • Amyotrophic Lateral Sclerosis / physiopathology
  • Animals
  • Antipyrine / analogs & derivatives
  • Antipyrine / therapeutic use
  • Biological Therapy / methods
  • Biomarkers / metabolism
  • Cell- and Tissue-Based Therapy / methods
  • Drug Design*
  • Drug Repositioning
  • Drugs, Investigational / therapeutic use*
  • Edaravone
  • Humans


  • Biomarkers
  • Drugs, Investigational
  • Edaravone
  • Antipyrine