International workshop - Preclinical testing in SMA 29-30th March 2010

One of the activities of TREAT-NMD is to harmonize experimental methods used to evaluate the efficacy of a test drug in animal models of disease and thereby reduce duplication of efforts and inconsistency of data that ultimately delay the progression of candidate molecules into the clinic. TREAT-NMD, in collaboration with the Wellstone Centres in the US, has already created a collection of standard operating procedures (SOPs) for animal models of DMD. TREAT-NMD partner organisations, Santhera and the University of Basel, recently organised a workshop to take a major step towards assessing this issue for mouse models of SMA with the aim to produce SOPs for use in the field of SMA research and to aid the assessment of new potential therapies in mouse models of SMA.

Thirty key SMA researchers convened last week in Zürich to discuss endpoint measurements in mice models and to reach a consensus as to which endpoints are particularly relevant to the clinical situation when assessing the preclinical efficacy of a candidate drug.

The meeting was also attended by four leading clinicians in the field and representatives from two patient organizations and industry. Moreover, it was agreed to form expert working groups extended to the wider community to compile standard protocols for each of the most important endpoints discussed at the workshop.

Once finalized, after receiving further input from the community, these protocols will be made accessible through the TREAT-NMD web site in the hope that the widespread use of standardized procedures will help increase the comparability of studies across laboratories. Both the level of expertise and the motivation of the attendees were impressive.

The workshop, organised and supported by TREAT-NMD, represents an important step towards a more efficient translation of preclinical research to the clinic in the process of drug development.

An extensive article about this workshop will be submitted for publication in Neuromuscular Disorders in the near future. For further information please contact Raffaella Willmann.