title: Guidelines for the preclinical in vivo evaluation of pharmacological active drugs for ALS/MND: report on the 142nd ENMC international workshop.
abstract: A transgenic animal model for anterior horn cell loss was established in 1994. This model is based on the insertion of a high copy number of disease-causing human Cu/Zn SOD mutations into the intact mouse genome. It serves to establish hypotheses for the pathogenesis of anterior horn cell death, but also to test potential pharmacological approaches to therapy in human ALS. Today, more than 100 -- published and unpublished -- compounds have been tested in this animal model, a large part of them being reported as successful. However, it proved to be difficult to translate these therapeutic successes in the animal model into human trials. Also, a number of disease-modifying strategies were difficult to reproduce, even by the same group. On the other hand, the step from mice to men means a huge investment for the sponsors of clinical trials and the scientific community. Therefore, establishment of standard methods for drug testing in ALS models is mandatory. In this workshop, clinical and preclinical researchers established in the field of ALS/MND met in Holland in March 2006 in order to establish guidelines for the community for drug testing in mouse models.
authors.affiliation: Department of Neurology, University of Ulm, Ulm, Germany. albert.ludolph@medizin.uni-ulm.de
authors.forename: Albert C
authors.initials: AC
authors.lastname: Ludolph
authors.forename: Caterina
authors.initials: C
authors.lastname: Bendotti
authors.forename: Eran
authors.initials: E
authors.lastname: Blaugrund
authors.forename: Bastian
authors.initials: B
authors.lastname: Hengerer
authors.forename: Jean-Philippe
authors.initials: JP
authors.lastname: Löffler
authors.forename: Joanne
authors.initials: J
authors.lastname: Martin
authors.forename: Vincent
authors.initials: V
authors.lastname: Meininger
authors.forename: Thomas
authors.initials: T
authors.lastname: Meyer
authors.forename: Saliha
authors.initials: S
authors.lastname: Moussaoui
authors.forename: Wim
authors.initials: W
authors.lastname: Robberecht
authors.forename: Sean
authors.initials: S
authors.lastname: Scott
authors.forename: Vincenzo
authors.initials: V
authors.lastname: Silani
authors.forename: Leonard H
authors.initials: LH
authors.lastname: Van Den Berg
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy*; D000818:Animals; D004195:Disease Models, Animal; D004353:Drug Evaluation, Preclinical / Q000379:methods*; D006801:Humans; D051379:Mice; D008822:Mice, Transgenic; D013482:Superoxide Dismutase / Q000235:genetics
chemical_list: D013482:Superoxide Dismutase