Back to Journals » Neuropsychiatric Disease and Treatment » Volume 5

Current and emerging treatments for amyotrophic lateral sclerosis

Authors Zoccolella S, Santamato A, Lamberti P

Published 3 November 2009 Volume 2009:5 Pages 577—595


Review by Single anonymous peer review

Peer reviewer comments 3

Stefano Zoccolella1, Andrea Santamato2, Paolo Lamberti3

1Azienda Ospedaliero-Universitaria

Ospedali Riuniti, Department of Medical and Neurological Sciences, Clinic of Nervous System Diseases, University of Foggia, Italy; 2Department of Physical Medicine and Rehabilitation, University of Foggia, OORR, Italy; 3Department of Neurology and Psychiatry, University of Bari, Italy

Background: Amyotrophic lateral sclerosis (ALS) is a relatively rare neurodegenerative disorder of both upper and lower motoneurons. Currently, the management of ALS is essentially symptoms-based, and riluzole, an antiglutamatergic agent, is the only drug for the treatment of ALS approved by the food and drug administration.

Objective: We reviewed current literature concerning emerging treatments for amyotrophic lateral sclerosis.

Methods: A Medline literature search was performed to identify all studies on ALS treatment published from January 1st, 1986 through August 31st, 2009. We selected papers concerning only disease-modifying therapy.

Results: Forty-eight compounds were identified and reviewed in this study.

Conclusions: Riluzole is the only compound that demonstrated a beneficial effect on ALS patients, but with only modest increase in survival. Although several drugs showed effective results in the animal models for ALS, none of them significantly prolonged survival or improved quality of life of ALS patients. Several factors have been implicated in explaining the predominantly negative results of numerous randomized clinical trials in ALS, including methodological problems in the use of animal-drug screening, the lack of assessment of pharmacokinetic profile of the drugs, and methodological pitfalls of clinical trials in ALS patients.

Keywords: amyotrophic lateral sclerosis, therapy, drug, survival

Creative Commons License © 2009 The Author(s). This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License. By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.