The SMA Trust is funding a new UK SMA Research Consortium for Spinal Muscular Atrophy in the UK over the next 3 years. The SMA Trust, founded in 2003, is the only UK charity solely focussed on funding research into finding a cure and treatments for Spinal Muscular Atrophy (SMA). 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 – making SMA the leading genetic cause of death in babies and toddlers.
The new UK SMA Research consortium is the first of its kind for SMA and is being announced on 29th September 2015 as part of the first ever UK SMA Awareness Week. It has come about because of impressive recent progress, leading to an ever-increasing need for research collaboration, not just within the SMA field, but in the overlap areas with 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 function throughout the lives of people living with SMA.
• Identify improved ways of delivering treatments in order to maximise benefit throughout the body.
The consortium will consist of world-class research/clinical experts based in Oxford, Edinburgh, London and Sheffield Universities. It will be led jointly by Professor Kevin Talbot (University of Oxford) and Professor Tom Gillingwater (University of Edinburgh). The intention is to form an initial nucleus that will, in time, draw together other leading UK scientists.
Last year The SMA Trust launched its Muscle Power Campaign, the aim being to raise funds which would enable us to:
• React quickly to fund projects which speed up clinical trials for new drugs
• Support UK Centres of Excellence
• Encourage collaboration and new research
• Help overall understanding of SMA
The new Consortium meets all these criteria and is exactly the type of project for which the fund was established.
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 comments: “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.”
To find out more detailed information about the research projects involved in the Consortium