Guidelines on Preclinical studies in ALS

Number 142
Date 24 April 2006

The 142nd ENMC International Workshop on “Preclinical Studies in ALS” organized by Caterina Bendotti (Milano) and Albert Ludolph (Ulm) was held in Naarden, Holland on March 24 - 26, 2006. Nineteen scientists from Europe, Israel and the U.S. participated in the workshop and discussed and established “Guidelines for the conduct of preclinical and proof of concept studies in ALS/MND models”.

The group decided to develop guidelines

I. since they felt that a number of current drug-finding studies in preclinical ALS/MND research are found to be unreliable by many in the field. The guidelines are developed to improve the methodology and the quality of the studies and consequently save resources for both, preclinical and clinical studies.

II. It was decided to consider “proof of concept” and “preclinical studies” separately. Aproof of concept study has the goal to elucidate the mechanism of the disease, may it be biochemical or physiological. Such a study may use a drug as an investigational tool. A preclinical study has the primary goal to develop a drug for use in humans; for these studies higher methodological standards must be met.

III. The group expressed the opinion that the current disease models are not satisfying since they only represent selected etiological factors of ALS/MND.  On the other hand the presence of transgenic disease models represents a unique advantage for the field to further develop long-awaited preclinical and clinical therapeutic strategies for this devastating disease. The group agreed that a major justification for the use of these disease models exists: they mirror important aspects of the pattern of vulnerability of ALS/MND.

The group agreed that it was the goal of the workshop that guidelines were developed in order to improve quality of the work without restricting individual groups without major resources. During the workshop the work was successfully completely and a draft consensus paper describing the guidelines for both proof of concept andpreclinical studies was developed and agreed on. Based on the draft, a full report will be written by the organizers, distributed to the participants of the workshop and discussed within the scientific ALS/MND community. Eventually the report will be published in a scientific journal which is widely read by the members of this community.

In a final effort, unmet needs of translational research (“mice to men”) were identified and suggestions were made to resolve these issues by individual working groups representing the necessary expertise.