The epidemiology and treatment of ALS: Focus on the heterogeneity of the disease and critical appraisal of therapeutic trials

Abstract

Effective treatments for amyotrophic lateral sclerosis (ALS) have remained elusive. Only riluzole, a drug thought to affect glutamate metabolism, improves survival albeit to modest extent. Explanations for the negative results of therapeutic trials include a likely heterogeneity, both in disease susceptibility and pathogenic mechanisms, and faulty methodology of clinical trials. Further understanding of these factors will lead to improvements in patient stratification, and in the design of future clinical trials.

Key words::

• Amyotrophic lateral sclerosis
• epidemiology
• diagnosis
• systematic review
• treatment

Acknowledgements

The study was supported by a grant from the Istituto Superiore di Sanità and the American ALS Association (ALSA). The research was supported in part by the Intramural Research Programme of the NIH, National Institute on Aging. The subject of the study has been discussed by EP in her Pharm D thesis.

Declaration of interest: The authors report no conflicts of interest. The authors alone are responsible for the content and writing of the paper.