Drugs in clinical development for the treatment of amyotrophic lateral sclerosis

ABSTRACT

Introduction: 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
• new drugs
• clinical trials
• cell therapy
• biomarkers

Article highlights

• Several new drugs are currently in clinical investigation for ALS thanks to recently public-private joint efforts.

• ALS patient’s associations are playing a critical role, as leader opinión and investors, in the fight against this severe disease.

• New ALS models for drug discovery and pharmacological screening are urgently needed.

• Drug repositioning is currently playing an important role to bring therapeutics for ALS patients.

This box summarizes key points contained in the article.

Acknowledgments

The authors would like to acknowledge Eoin Comenford from Dublin Institute of Technology for his editing and writing assistance.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Additional information

Funding

This work was financially supported by MINECO (Grant no. SAF2012-37979-C03-01 to A. Martinez and IJCI-2014-20767 to V. Palamo).