making muscle wasting history

A very good book you should read is 'Bad Science' by Dr. Ben Goldacre. Dr. Goldacre looks into specifically the claims of alternative medicine and the big pharmaceutical companies and especially trials.

Now the usual 'guff' about research on a lot of these MD websites just links to specifically one trial or two thats often impenetrable to most. It may show positive results or talk about yet another ruddy mouse.
In Dr. Goldacre's book he breaks things down so you understand what exactly trials are about.

He explains why trials should be randomized (to aviod bias), prefrably double blind (neither participant nor doctor know what treatment is being undertaken) and whether a placebo or another 'treatment' is used and also the correct interpretation of the statistics. It's helped me reappraise trials I now read. Find his book here- http://www.amazon.co.uk/Bad-Science-Ben-Goldacre/dp/000728487X/ref=sr_1_1?ie=UTF8&s=books&qid=1282866707&sr=8-1
or ebook here http://ebooks.whsmith.co.uk/7E381C30-8DA9-4199-84B3-102CE4DE36CB/10/132/en/ContentDetails.htm?ID=E6646D8D-36F5-4559-A432-FED87AF6860B

Ok now to the main point of my blog, Meta Analysis. Meta analysis of medical trials in particular which you can and should read at the Cochrane review. How it works is thus, lets use steriods as an example, every trial of steriods in boys/men with DMD thats ever been conducted is reviewed by two different professional reviewers who only look at, whether its a fair trial and what all data means for the drug in question and whether it met it's criteria. So to conclude this part steriods were found to beneficial at 0.75/mg/kg/day of prednisolone. Read this here - http://www2.cochrane.org/reviews/en/ab003725.html

Now the beauty of a meta analysis is that its totally unbiased, they evaluate good trials (positive results) and bad trials (negative results) and present their conclutions in plain english too. Please do explore the Cochrane reviews they are very interesting.

The only frustrating part is the lack of trial data the reviews can evaluate on specific DMD 'treatments', I can see on the site they will investigate exon skipping, utrophin etc, but this is only after there's been HUMAN trials.

Dr. Goldacre pleasingly agrees with us that you can't tell much with mice and cells in a dish in a sterile lab. We all have heard of our MDx mouse but it's only to see if any new drug is toxic mainly and if it shows any affect on DMD. All other wild claims excited scientists waft around like fine silk should be taken with a hefty pinch of salt, getting to a phase I/II trial in man is far more important as with all subsequent human trials. These can be meta analysed then, to see if that 1 awesome drug (God willing) has passed the tests and gained wide range usage like steriods.

Now many might think 'meta analysis' who care's Ian! Well we should all care because say a drug company gave us a drug but 'mislaid' some bad vital evidence that started giving cancer to boys/men who thought they were gunna get better. Also it's a great way to to see how much more research needs doing or needs speeding up.

Ok to highlight what differences in research between DMD and other diseases I saw, I thought I'd compare with some rough figures I found between DMD and say another devasting disease like Motor Neuron Disease/ALS.

Ok the numbers I counted include every trial into specific drugs/treatments for each disease. I excluded secondary management and symptom control. It was just on things aimed at halting each disease.

DMD first, I found a total of 4 meta analysis studies, 2 had figures and 2 are in so-called 'protocol' stage, which means their next up to be analysed in the near future. Ok of the two Meta analysis with figures they included a total of 10 trials and over 356 participants.

I thought that sounds pretty reasonable, I know they are based on current drugs, the whole raft of Ataluren, Exon skipping, Utrophin and all the other trials will be analysed soon i'm sure. It goes to show that we need less mouse trials and more human trials especially considering the average two deaths a week from DMD. There's unfortunately YEARS more before things get properly evaluated.

Now on to MND/ALS I found a total of 10 meta analysis studies, 6 had figures 1 had no figures and 3 are in so-called 'protocol' stage, which means their next up to be analysed. Ok of the 6 meta analyis with figures they included a total of 30 trials and over 5185 participants.

Thats a stark difference to DMD even including the 1-5 in 100,000 incidence rate in MND which means MND is slightly rarer. How can a slightly rarer disease get many more trials baffles me, I feel we should be at roughly the same amount of research.

Well it just shows you the utter lack of attention DMD got in the twenty years prior to 2001 (when thank God Action Duchenne helped raise awareness and kick started the MDEX consortium).

All this means is, come on science speed up your ideas and human trials in Humans and rely less on your favourite mdx mouse. Lets match other disease research sooner rather than later.

By the way none of this is having a go at scientists I and all others with DMD thank you for busting a gut and looking at getting rid of or a least slowing DMD to a standstill. Bear this in mind however no more kids/adults with DMD need to come to a standstill DMD DOES.


Thanks for reading.