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

Albert C. Ludolph, Caterina Bendotti, Eran Blaugrund, Bastian Hengerer, Jean Philippe Löffler, Joanne Martin, Vincent Meininger, Thomas Meyer, Saliha Moussaoui, Wim Robberecht, Sean Scott, Vincenzo Silani, Leonard H. Van Den Berg

Research output: Contribution to journalArticle

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 Today, more than 100 - published and unpublished - compounds have been tested in this animal model, a large part of them being reported as successful.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

Original languageEnglish
Pages (from-to)217-223
Number of pages7
JournalAmyotrophic Lateral Sclerosis
Volume8
Issue number4
DOIs
Publication statusPublished - 2007

ASJC Scopus subject areas

  • Clinical Neurology
  • Neurology

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