Finding a cure or a treatment for all the different kinds of CMT is pretty much like the Holy Grail for most of us, isn't it? Living with an incurable condition isn't the easiest thing in the world. So research is a major priority for us, and indeed for all the CMT organisations around the world.
Opportunities to fund projects that we can afford don't come up very often, and like buses - when they do, three come along at once...!
Opportunity Number 1
We are delighted to have been asked to partner with the Muscular Dystrophy Campaign on a basic science project at Imperial College London.
The project is looking into cell repair mechanisms into a very specific and rare type of CMT, and whilst it probably won't have any direct impact on you living with CMT anytime soon, any research which can fill in the gaps in knowledge into how the nerve cells work adds to the overall pool of knowledge and ultimately, may well help in the development of treatments in the future. The project is using brand new scanning techniques, which allows the researchers to study live nerve cells, both healthy (controls) and those with this particular type of CMT.
The cost of the project is £77,600 over three years, and we'd like to contribute half of the cost, ideally - ie, just under £13,000 per year.
Opportunity Number 2
Whilst the basic science work above is vital, research is also required into treatment interventions that could have a positive impact on people with CMT now, such as physiotherapy treatments or orthotics etc. Yes, we need a "cure" - but we also have to live with the effects on a day to day basis.
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Gita in action at our Conference in April.
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Balance issues and falls are probably the worst issues that we have to face, and if there was a proven intervention that could help this, I'm sure we'd all jump at the chance. Dr Gita Ramdharry-Jackson has applied to us for funding for a one year pilot project, looking into various interventions that could help address balance issues. If the pilot proved successful, she'd like to undertake a much larger scale trial to confirm her data.
She is applying for sufficient funds to employ a research physiotherapist to educate, train and monitor a group of participants (who would probably be drawn from the cohort of patients currently seen at the Centre for Neuromuscular Disease in London) in a variety of exercises and techniques that, it is hoped, may improve their balance and core stability.
The cost of this one year pilot project is £32,000.
MRI Scanning Project
On top of these new projects, it mustn't be forgotten that we are already committed to providing the Centre for Neuromuscular Disease with £10,000 per year (we're entering year 2), to continue their innovative MRI scanning project, a project that is providing objective proof of the progression of CMT, by measuring the fat that infiltrates into muscles as they atrophy. In future years, this will be a vital outcome measure for clinical trials, giving absolute confirmation of the efficacy (or not) of any treatment.
WE NEED YOUR HELP URGENTLY
The problem we have is that, ideally, we want to fund all three projects, but the Research Fund stands at £44,000 at this moment in time.
It doesn't take a mathematician to calculate that we have a shortfall of £11,000 for this year, and of course, a further £23,000 would be needed next year, and more the year after! In the future, once we've recruited a new Fundraising Manager, we would hope that the Research Fund will get lots of lolly to ensure that we can fund more and more of this kind of research, but at the moment, we just don't have the funds available. And it's not possible to divert funds from elsewhere - obviously monies donated for other specific projects such as the Young People's Weekend HAS to be used solely for that, and the General Fund is also looking poorly, with monies committed to the update of the new edition of our book, and all the rest of the day to day expenditure. (Don't get us wrong, we're not in trouble... just tightening belts a little, til New Fundraiser Bod is recruited and doing his/her stuff!)
Can you help? We know you're an amazingly generous lot, and we KNOW that funding research is as important to you as it is to us.
If you can help, click on the button below to make an instant donation to the Research Fund - or you can send a cheque to the usual address, or text DOSH14 £10 to 70070.
