Dr Claire Wood is aiming to gain a better understanding of the bone and growth problems associated with Duchenne muscular dystrophy. She will assess the bone health and growth of mouse models of Duchenne muscular dystrophy and test whether this can be improved by treating the mice with growth-promoters.
Dr Wood has been awarded a four year Medical Research Council (MRC) Clinical Research Training Fellowship, which is being co-funded by Muscular Dystrophy UK. Clinical Research Fellowships are training programmes that help young talented clinicians to pursue academic research careers in the neuromuscular field. Clinical fellows, with one foot in the laboratory and one foot in the clinic, are ideally placed to promote translational research.
What are the researchers aiming to do?
Growth problems and the weakening and thinning of bones (osteoporosis) are common in people with Duchenne muscular dystrophy. It is not known whether these bone and growth problems are a result of the muscle weakness (our bones need to be ‘loaded’ by muscle activity in order to strengthen and grow efficiently), or whether growth signals in the body are being disrupted.
In this project, Dr Wood will use three different mouse models of Duchenne muscular dystrophy to gain a better understanding of what causes bone and growth problems associated with the condition. This will be the first time that one of these models has been studied in terms of its bone and growth characteristics. It is very important to learn more about this mouse model in order to assess whether it is suitable for using in other experiments.
Dr Wood will measure a variety of features related to bone health and development and compare these measurements to those from healthy mice. For example, she will measure the length and fragility of certain bones, as well as the overall growth of the mice i.e. their weight and tail/body lengths. She will also grow bones from the feet of the mouse embryos to see if they also show poor growth. This will tell us if there is a primary problem relating to the skeleton, in addition to the problems caused by steroids and muscle weakness.
Steroids are currently the only treatment available for Duchenne muscular dystrophy but they have side effects, including an increased risk of growth problems and osteoporosis. Dr Wood will assess the effect that steroids have on bone health and development by giving the mouse models daily injections of steroids over a four week period and then repeating the bone measurements described above.
Finally, Dr Wood will investigate whether growth-promoters could be a potential treatment for the bone and growth problems associated with Duchenne muscular dystrophy. She will test this by giving the mouse models daily injections of growth-promoters over a four week period and then repeating the different bone measurements.
How will the outcomes of the research benefit patients?
Although this research is being carried out on mice, it will give a valuable insight into the bone and growth problems of people with Duchenne muscular dystrophy. It will also give scientists and clinicians more information on how steroids can affect the bones, which could be important for a range of conditions where steroids are prescribed.
Osteoporosis increases the chance of bones fracturing, which can have a lasting effect on walking ability (ambulance) and pain experienced by people with Duchenne muscular dystrophy. Improving bone health and reducing the risk of osteroporosis is therefore very important in prolonging ambulation and ensuring a good quality of life for people with the condition. This project is testing whether growth-promoters could improve bone health and development and improve growth problems; if the results from the mouse models are positive, then this could lead to a clinical trial testing growth promoters in people with Duchenne muscular dystrophy.
Project leader: Dr Claire Wood
Location: University of Edinburgh & Newcastle University
Supervisors: Professor Colin Farquharson (University of Edinburgh), Professor Faisal Ahmed (Glasgow University) and Professor Volker Straub (Newcastle University)
Conditions: Duchenne muscular dystrophy
Duration: four years (part time research), starting 2016
Total project cost: £284,774.25 (£100,000 contribution from Muscular Dystrophy UK)
Official title: Characterisation of skeletal development and the use of anabolic agents in murine models of Duchenne muscular dystrophy
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