What research does the MDA fund?

Muscular Dystrophy Australia funds cutting edge research at the National Muscular Dystrophy Research Centre (NMDRC) which is situated within the Murdoch Childrens Research Institute in Parkville, Victoria. In excess of half a million dollars is invested each year as part of MDA’s philosophy to invest equally between supporting the muscular dystrophy community today in their everyday lives, and research to find a treatment in the future.

Boris M Struk (MDA Executive Director), Kristina Elvidge (MDA Scientific Communications Officer) and Jason White (Senior Scientist at NMDRC) in the lab discussing NMDRC research projects

Which muscle conditions are studied?
The research team at the NMDRC is working to find treatments for many of the muscular dystrophies (MD) and related neuromuscular conditions. The research generally starts with Duchenne MD since it is the most common MD affecting children and model systems are readily available to study it. However, it is a key aim that any treatments found also be applicable to other types of MD.

What is the strategy to find treatments?
It is anticipated that there won’t be just one ‘magic pill’ treatment for MD so a combination of treatments will be needed to attack the problem from all angles. The two main strategies being pursued at the NMDRC are detailed below and these could be combined for an even better result. They could also be used to further boost the effectiveness of other promising treatments currently being developed such as gene therapy, exon skipping for Duchenne MD or antisense technology for myotonic dystrophy.

Fighting inflammation
A common feature amongst many of the MDs is that the muscles become inflamed which is damaging to muscle. So one of the research strategies being pursued is to find ways to reduce the inflammation — this would help the muscles stay stronger for longer.

Currently the only drug treatment that has been proven to keep boys with Duchenne MD walking for longer is corticosteroids (such as prednisolone). They work, at least in part, by reducing inflammation in the muscle. However, taking corticosteroids constantly for a long period brings unwanted side effects. This research hopes to find an anti-inflammatory treatment that would work at least as well as steroids, if not better, with fewer side effects.

Repairing muscles
In MD, the body is initially quite efficient at repairing damaged muscles and muscle weakness is kept at bay, but eventually it can’t keep up and the muscles start to weaken. So another arm of the research strategy is to improve the ability of the body to repair damaged muscles. In the laboratory this involves studying how stem cells regenerate muscle and exploring ways in which this process could be enhanced.

How are potential treatments identified?
One source of potential drugs to test in the lab are those that already exist for treating other conditions. For example, there are many drugs available or in clinical trial for conditions such as arthritis which reduce inflammation.

Secondly, there are so called ‘libraries’ of substances that have the potential to treat disease. These libraries of hundreds or thousands of substances can be screened for any positive effect on the muscles.

Another avenue is to keep an eye out for molecules being discovered by scientists around the world that have properties that could in theory counteract the effects of MD. These can then be tested in the laboratory to find out if they work in practice. Sometimes these molecules aren’t synthetic drugs but natural substances isolated from foods and given as a dietary supplement, for example certain vitamins given at high doses. These are called ‘neutraceuticals’ and are promising because they might offer a way to manage or prevent the symptoms of a condition without harmful side effects.

To find out if a substance from any of these sources shows promise for treating muscular dystrophy, they are tested in model systems in the laboratory. Well-established models of Duchenne MD are available, along with reliable methods for studying them. Several breeds of mice that have conditions similar to Duchenne MD are used, as well as muscle cells from MD patients.

Substances are added to muscle cells grown in the laboratory and any positive effect monitored. Promising candidates are then administered to mouse models of MD and the strength of the muscles measured and compared to mice that have not received the treatment. Whether the muscles are being protected from damage can also be assessed by looking at the muscles under a microscope.

Would this research be possible without the MDA?
MDA has provided research funding since 1994 and this consistent income has provided a solid foundation for the centre to grow and explore research avenues that would not have otherwise been possible. The funding goes towards salaries for key senior scientists and scholarships for PhD students. Importantly it also provides funds for buying equipment and laboratory consumables.

Dr Jason White, Senior Research Fellow at the NMDRC said “One of the focuses of the funding has always been to train the next generation of scientists that provide the future of MD research. This is a very important aspect that would not be possible without the ongoing support from MDA”.

What about clinical trials?
MDA is keen to ensure that the MD community of Victoria doesn’t miss out on clinical trials of any new potential treatments. As such we employed an MDA Neuromuscular Coordinator (Daniella Villano). Besides her role running the clinic at the Royal Children’s Hospital, she coordinates the enrollment of patients into the clinical trials currently underway at the hospital. Without Dani these clinical trials would not be possible. Find out more about clinical trials.

Further information
• New research grant to test possible MD therapy at the NMDRC
• More research news
• You can get regular updates by becoming a friend of the MDA Facebook page

If you have any questions, please contact us:
Phone: (03) 9320 9555

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Updated 11 August 2014




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