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The discovery of future neuro-therapeutic molecules

Final Report Summary - NEUROSCREEN (The Discovery of Future Neuro-therapeutic Molecules)

Described more explicitly, the NEUROSCREEN project aimed to maximise the utility of new rodent and human (NS) cell lines in the design of unique assays for the tractable automated discovery of small molecules that modulate the biological function of neural stem cells and derivative neurons and glia. Such a chemical genetics approach applied to scalable cell lines was expected to yield molecular discoveries with inherent attributes of value for their prospective development as future regenerative medicines to treat neurological and neurodegenerative diseases.

The main achievement is the transfer of knowledge from science base to application. 'If Europe is to benefit, excellence in the science base is not enough: it is essential to have the capacity to translate knowledge into new products, processes and services, which in turn will generate benefits to society, skilled jobs and prosperity' (from LifeSciHealth priority, 'Lifesciences and biotechnology: a strategy for Europe'). The success of any knowledge-based economy rests upon the generation, diffusion and application of new knowledge and the work here described moves strongly from the traditional academic focus on understanding stem cells to a comprehensive commercial focus, an essential move for the transfer and application of new knowledge.

The establishment of a set of procedures to check the quality of the neural stem cells and a proper storage facility can be the starting point to generate a consistent source of biological material. The standardisation of all processes, the tracking system and the study of the administrative / legal aspects helped identify a specific and successful workflow to be applied to the management of a stem cell bank and to the distribution of highly validated cells. Without proper visibility and diffusion, information and reagents originated by the NEUROSCREEN project will be of limited use.

Further results generated suggest that there is commercial potential for compounds to be used as cell culture media additives based on demonstrated compound stability. This potential represents the most immediate commercial outcome for the consortium since only a modest amount of further development work would be required to validate commercial utility. This is in contrast to the extensive efforts that would be required to progress compounds through the drug discovery process. A key challenge in stem cell research is the maintenance and modulation of stem cell populations. In most cases, this is currently achieved through the use of complex and expensive cocktails of biological reagents.

The use of small molecules has already been demonstrated to substitute for biological signalling factors for the maintenance of mouse embryonic stem cell populations and so it remains a commercially attractive proposition to pursue the chemical compounds that, either singularly or in unique combinations, may confer similar maintenance properties to the numerous other stem cell populations. In addition to increasing reproducibility and standardisation across research groups, the development of new cell culture media using small molecules to substitute for biological signalling factors is also likely significantly to reduce the costs of cell culture reagents, thereby serving the international research community as a whole.
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