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In September 2008 the Myotubular Trust put out a call for grants and after a rigorous application and peer review process their Scientific Advisory Board recommended that the Trust support two applications, both of which will begin in September 2009. The first project, which will be run by Dr Richard Piercy, a Vet at The Royal Veterinary College, will use a naturally occurring Labrador dog model for the disease, to try and understand what aspect of muscle function has stopped working properly in these muscle conditions. To do this, he is going to take muscle cells from both a healthy and an MTM-affected dog, grow them in the laboratory and compare these cells for differences in cell pathology, such as cell size and shape. He will also compare the muscle cells for how well they work when asked to contract. This will give an indication of the strength of the muscle. The second project, which will be run by Dr Anna Buj-Bello at INSERM, a French laboratory, will use mouse models for the disease. She will try a variety of therapeutic 'rescue' approaches such as gene therapy and drug administration. Her mouse models lack myotubularin either from just muscle or from all body tissues. She will then add back myotubularin, or a drug, and monitor all of the body organs over time for health improvements. She will treat mice both prior, and subsequent to, the development of the clinical symptoms of MTM. Therefore these experiments will not only tell us whether this treatment will be suitable to alleviate the clinical symptoms of the disease, but also whether it could stop the development of the disease from the outset. The research output from both of these projects is likely to suggest therapeutic strategies to treat MTM patients of all ages. The full lay report can be viewed on the Myotubular Trust website. A second Call for Grants will take place this September meaning that further research can begin in September 2010; bringing ever closer the day when a cure, or treatment, can be found. Read about the stages, which have led to the announcement.
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