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

Ettore Beghi (1) , Adriano Chiò , Philippe Couratier (2, 3) , Jesùs Esteban , Giancarlo Logroscino , Andrea Millul , Douglas Mitchell , Pierre-Marie Preux (3, 4, 5) , Elisabetta Pupillo , Zorica Stevic , Robert Swingler , Bryan J Traynor , Leonard H van den Berg , Jan H Veldink , Stefano Zoccolella , Benoît Marin (3, 4, 5)

1 Laboratory of Neurologic Diseases, Mario Negri Institute, Milan
2 Service de Neurologie [CHU Limoges]
3 NETEC - Neuroépidémiologie Tropicale et Comparée
4 SIME - Service de l'Information Médicale et de l'Évaluation [CHU Limoges]
5 Laboratoire de Biostatistique et d'Informatique Médicale

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.

Domaines: Sciences du Vivant [q-bio]; Santé publique et épidémiologie

Ettore Beghi, Adriano Chiò, Philippe Couratier, Jesùs Esteban, Giancarlo Logroscino, et al.. The epidemiology and treatment of ALS: focus on the heterogeneity of the disease and critical appraisal of therapeutic trials.. Amyotrophic Lateral Sclerosis and Other Motor Neuron Disorders, 2011, 12 (1), pp.1-10. ⟨10.3109/17482968.2010.502940⟩. ⟨hal-00605454⟩