New £1.3m research consortium targets spinal muscular atrophy | University of Oxford

New £1.3m research consortium targets spinal muscular atrophy

2 October 2015

Oxford University will lead a new collaborative initiative for Spinal Muscular Atrophy (SMA) research in the UK over the next 3 years. SMA is the leading genetic cause of death in babies and toddlers.

SMA is caused by a faulty gene that affects nerve cells needed to control the muscles we use for moving, swallowing and breathing. 1 in 40 of us is a carrier and in the most severe cases (50% of those affected), children rarely live beyond 2 years old. The programme is funded by the SMA Trust, founded in 2003 as the only UK charity solely focussed on funding research into finding a cure and treatments for Spinal Muscular Atrophy.

The new UK Research consortium, the first of its kind for SMA, was announced as part of the first ever UK SMA Awareness Week. It has come about because of impressive recent progress in research that has highlighted the increasing need for research collaboration, not just within the SMA field, but in areas common to other neuromuscular conditions like Muscular Dystrophy and Motor Neurone Disease.

The main aims are to further develop existing drug targets and identify new neuroprotective therapies to maintain nerve function throughout the lives of people living with SMA, and to identify improved ways of delivering treatments in order to maximise benefit throughout the body.

The consortium will consist of world-class research and clinical experts based at Oxford, Edinburgh, London and Sheffield Universities. It will be led jointly by Professor Kevin Talbot from the University of Oxford and Edinburgh University's Professor Tom Gillingwater. The current team are an initial nucleus that it is hoped will draw together other leading UK scientists in time.

Professor Kevin Talbot said: ‘By bringing together specialists across the UK, the UK SMA Consortium will facilitate greater integration between research groups in order to maximise the impact of our research on accelerating the identification of effective therapies for SMA.’

The SMA Trust has spent nearly £3m since it was founded and now funds 75% of all UK charity funded research into the condition. It also helped create SMA Europe, an umbrella organisation that funds research projects all over Europe and the US, as well as promoting knowledge-sharing, collaboration and representation of a united ‘patient voice’ to pharmaceutical companies and regulatory authorities involved in clinical trials.

Joanna Mitchell, CEO of The SMA Trust said: 'These are exciting times in SMA research and for the new UK research consortium. Recent years have seen rapid advances, with several companies now actively pursuing clinical trials for potential treatments. Whilst a cure for SMA remains the ultimate goal, we also recognise that, for the time being, ‘success’ is equally likely to come in the form of combinations of treatments that improve the quality of life of people living with SMA.'

For more information or to request an interview contact Tom Calver at Oxford University on 01865 270046 or news.office@admin.ox.ac.uk.

A video of Professor Kevin Talbot explaining about SMA research is at: https://www.youtube.com/watch?v=U1F-vGpyeMQ