Source: Muscular Dystrophy Campaign press release
Professor Dame Kay Davies, Professor Matthew Wood and Dr Angela Russell will receive the grants for their research projects to improve understanding of or explore potential treatments for Duchenne muscular dystrophy. The grants have been awarded by charity the Muscular Dystrophy Campaign and fellow members of the Duchenne Forum, and will be awarded over a two or four-year period.
One hundred children are born in the UK each year with Duchenne muscular dystrophy. The condition, which almost always affects boys, is caused by the lack of a vital muscle protein and causes muscles to weaken and waste over time, leading to increasing and severe disability. It affects not only the muscles of the limbs, but also those of the heart and lungs, and few of those born with the condition will live to see their 30th birthday.
Professor Dame Kay Davies’ project will build on her previous work attempting to increase levels of a muscle protein which may compensate for that which is lacking in people affected by Duchenne muscular dystrophy. One drug developed for this purpose by Professor Davies and her colleagues is already in clinical trial. Dr Russell’s work will complement Professor Davies’ work and will seek to better understand how this potential treatment could work. Professor Wood will identify ‘biomarkers’, biological indicators that can be used to measure the progression of Duchenne muscular dystrophy, and a patient’s response to future treatments during clinical trials.
The grants are among seven being awarded by the Duchenne Forum, a collaboration established between six charities to accelerate progress in the search for effective treatments and cures for Duchenne muscular dystrophy. The Duchenne Forum charities are the Muscular Dystrophy Campaign, Alex’s Wish, Duchenne Children’s Trust, Duchenne Research Fund, Joining Jack and Harrison’s Fund.
Dr Marita Pohlschmidt, Director of Research for the Muscular Dystrophy Campaign, said: “Following the dedicated fundraising efforts of our supporters, we are delighted to be able to back the vital work of these three highly respected scientists. Duchenne muscular dystrophy is a devastating condition and we will continue to fund a comprehensive programme of research until treatments are found for every single person affected.”
Professor Dame Kay Davies said: “This grant makes a big difference to us as it allows the continued screening of the cell line we developed with previous funding from the Muscular Dystrophy Campaign. This approach, which is being carried out in collaboration with Dr Angela Russell in the Chemistry department of Oxford University, will allow us to identify novel hits which increase utrophin levels which we can develop as drug leads.”
Professor Matthew Wood said: "We are very grateful that the Muscular Dystrophy Campaign has decided to support this PhD project which looks at the levels of microRNAs for Duchenne muscular dystrophy. These microRNAs are short pieces of RNA which may be detected in blood. Certain microRNAs change following treatment so we want to use them as biomarkers as it provides an easy and non-invasive way of looking at how well a treatment is working. We are hoping to expand on this and find even better biomarkers."