Stuff that occurs to me

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Think of this blog as a sort of nursery for my half-baked ideas hence 'stuff that occurs to me'.

Contact: @JoBrodie Email: jo DOT brodie AT gmail DOT com

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Saturday, 1 September 2012

Duchenne, petitions and research funding infrastructure

The other day someone on Twitter asked me to sign a petition calling on the Government to fund research into a cure for Duchenne Muscular Dystrophy (DMD). Her young son has the condition, it's incurable and results in muscle wasting but mind sparing - so you know what you're going through and that it's fatal. Grim. She wants to find a cure for her son which is very understandable but I'd be very surprised if research results produced now or in the next few years would result in a cure in the right timescale.

I'm all for curing Duchenne and I like signing petitions but I don't think that this petition will necessarily help, I'm not even sure that it can.

As far as I'm aware the Government contributes funding for medical research through the MRC (Medical Research Charity) which is one of seven research councils, all of which have responsiblity for different areas of science, medicine, technology and other research funding. 

The MRC has definitely funded research into DMD, so by extension the Government has funded research into DMD. I don't know if you can say that it was particularly directed at curing DMD though. An awful lot of basic research needs to be done first in order to understand a condition and it might be difficult to know, depending on how much is already understood about DMD, which avenue holds most promise. 

We've still not cured diabetes and it's been known about since antiquity (earliest written record believed to be the Ebers Papyrus). Insulin's been available to keep people with Type 1 diabetes alive since the 1920s and we now understand a great deal about the mechanisms underlying Type 1, Type 2 and other forms of diabetes and treatments are much better tailored. We're able to 'tinker' with diabetes and, in some cases, slow the progression to full-blown Type 1 diabetes, reduce the amount of insulin someone with Type 1 needs, normalise blood glucose levels in someone with Type 2 diabetes - but these are all fairly extreme cases requiring surgery or immune suppression. And generally these aren't thought of as a long-term permanent cure, although I often hear it called that for bariatric surgery.

If the Government is already funding Duchenne research, indirectly, and this petition won't help (because (a) the Gov wouldn't specifically fund Duchenne and (b) it's perhaps a bit of a challenge to pitch funding directly at a cure for any condition) - what would?

Possibly supporting charities that fund research into Duchenne might help, perhaps getting on the research decision-making panel (if possible) to have a say in the direction of research, seeing what clinical trials might be available and perhaps raising the issue in Parliament (mostly to get news coverage I expect, rather than funding). It's also worth seeing what people are saying about the condition on social media, in terms of hearing about news and events.

Charities
According to the Charity Commission there are several Duchenne charities operating in England and Wales. Some provide patient & carer advice and support and some fund medical research. The ones highlighted fund research.

The ones I found are

1101971 ACTION DUCHENNE LIMITED     Registered
328220 DUCHENNE FAMILY SUPPORT GROUP     Removed
1120873 DUCHENNE MUSCULAR DYSTROPHY TRUST   Registered
1146355 DUCHENNE NOW                     Registered
1147094 THE DUCHENNE CHILDREN'S TRUST     Registered
1128653 THE DUCHENNE FAMILY SUPPORT GROUP     Registered
1119068 THE DUCHENNE RESEARCH FUND             Registered

Not sure what it means by the second one being 'removed' as their website still seems operational and has the charity number listed on its site.
  • Action Duchenne: Website | Twitter
  • Duchenne Muscular Dystrophy Trust: Website (incidentally, gorgeous creepy ambient music)
  • Duchenne Now: Website
  • The Duchenne Research Fund: Website 
Not to mention there are several charities for Muscular Dystrophy more generally.

Clinical trials
Both these websites have information on registered clinical trials - I don't know if there's a research network for Duchenne (in the same way that there are for cancer and diabetes). There's a Duchenne Alliance of a variety of Duchenne support groups though.

ClinicalTrials.gov
Controlled-Trials.com

Who's mentioned it in Parliament?
There was an Early Day Motion back in July 2012 - Duchenne Muscular Dystrophy (No. 2) - about the showing of a film in the Atlee Room, the event seems to have been coordinated by Muscular Dystrophy Campaign and Action Duchenne.

This link will run a search for mentions of Duchenne:
http://www.theyworkforyou.com/search/?s=duchenne

Social media
Twitter: This link will return a list of tweets recently posted about Duchenne
https://twitter.com/#!/search/realtime/duchenne

Topsy is like a supercharged search for Twitter:
http://topsy.com/s?q=duchenne from which I discovered this news story about a newly trialled drug called Eteplirsen. To be honest I'd like to read the original published paper (and probably get some help in understanding its nuances) before I'd be as cheerful as the mum in this story.

Undoubtedly there are Facebook groups too.

3 comments:

  1. I think that you have missed a number of young but very active charities specifically raising money for research.
    Harrison's Fund www.Harrisonsfund.org
    Joining Jack www.joiningjack.org
    there are a number of others.

    ReplyDelete
  2. Thanks Jenni that's great, I had missed those. I just searched the Charity Commission's page for 'Duchenne' so it seems a same that they only return hits that contain that word in the charity's title. Might suggest that they amend their search algorithm to accommodate keywords as well as titles.

    ReplyDelete
  3. Dear Jo,this is Andy. I work for Action Duchenne and noticed your blog pop up on my google alerts this morning. If you'd like more information on research for DMD then you can check the research part of our website here: http://www.actionduchenne.org/action.

    You're absolutely right that the Government has funded, via the MRC, £2.75million on DMD in 2010/11. There is also £9.3million over 5 years set aside specifically for a research programme on on the molecular basis of childhood diseases. This does not mean that it is futile to request more however - especially given the advances being made. In all of these situations, it's important to make your case for funding and keep pressure on.

    It is also important that we do not neglect other areas aside from research. Young people living with Duchenne are living longer, but often see their clinical and social support drop off once they leave paediatric care. Care standards also differ widely throughout the UK - especially in comparison to places like Denmark, where people are living into their fifties. These are areas that must not be forgotten when discussing Duchenne.

    It's great to see you taking an interest though and if you'd like to discuss anything further then please contact me at andy@actionduchenne.org.

    ReplyDelete

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