Guidelines for the preclinical in vivo evaluation of pharmacological active drugs for ALS MND: Report on the 142nd ENMC international workshop

• Published in: Amyotrophic lateral sclerosis Vol. 8; no. 4; pp. 217 - 223
• Main Authors: Ludolph, Albert C., Bendotti, Caterina, Blaugrund, Eran, Hengerer, Bastian, Löffler, Jean-Philippe, Martin, Joanne, Meininger, Vincent, Meyer, Thomas, Moussaoui, Saliha, Robberecht, Wim, Scott, Sean, Silani, Vincenzo, Van Den Berg, Leonard H., ENMC GROUP FOR THE ESTABLISHMENT OF GUIDELINES FOR THE CONDUCT OF PRECLINICAL AND PROOF OF CONCEPT STUDIES IN ALS MND MODELS
• Format: Journal Article
• Language: English
• Published: England Informa UK Ltd 2007; Taylor & Francis
• Subjects: Amyotrophic Lateral Sclerosis - drug therapy; Animals; Disease Models, Animal; Drug Evaluation, Preclinical - methods; Humans; Mice; Mice, Transgenic; Superoxide Dismutase - genetics
• ISSN: 1748-2968; 1471-180X
• DOI: 10.1080/17482960701292837

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 Today, more than 100 - published and unpublished - compounds have been tested in this animal model, a large part of them being reported as successful.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.