Why is Duchenne not included in inherited Cardiomyopathy
I came across a stem cell trials for Cardiomyopathy, and find that there are lots of trials going on to find a cure for Cardiomyopathy. I tried to find out if there is any DMD patient included in the trials for Cardiomyopathy; it turns out that DMD is not defined in the list of inherited disorders which lead to Cardiomyopathy.
Cardiomyopathy is the reason why this disease is 100% fatal. Does anyone know why DMD is not cosidered for these trails on Cardiomyopathy.
Replies to This Discussion
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Permalink Reply by Deb Robins on
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I agree with you about the relatedness of Duchenne to Cardiomyopathy because if you check out this Human Diseasome it shows visually the genetic relatedness of diseases (zoom in on Cardiomyopathy) I find it interesting that Duchenne has more in common and is closer in relationship to Cardiomyopathy than any of the many separate muscle disorders, which DMD is traditionally lumped in with. But perhaps it isn't a good idea to be in a trial if one has two diseases because one of the DMD scientists replied to a question from a parent the other day about a similar thing - being included in a DMD trial if one is affected by another genetic disorder on top of DMD (as her son has two genetic conditions)... She is a scientist rather than a clinician choosing trial candidates, so she couldn't give a definitive reason but she guessed that a person may not be considered for a trial if there is a possibility that the drug might be treated differently by the body due to the second disease. E.g. the drugs are longer or shorter in the body, or maybe are cleared in a different way. She said that having two diseases may cause the researchers to draw wrong conclusions. That is not to say that once something is proven efficacious that one wouldn't be able to use the treatment just because one has another condition. The doctors would just have to monitor the person more carefully given the presence of the second disease. I have only related a possible answer given to one of our parents recently - I don't know for sure.
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Permalink Reply by Tulika on
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The Doc asks the exact same question in the news article
"if the diseases are similar in some molecular pathways, perhaps the heart attack drugs should be tested against muscular dystrophy. "
I can understand drug companies trying to de-risk their trails by not including DMD. Even if companies wanted to they are not allowed to include DMD patients in Cardiomyopthy trials. Problem is with World Health Organization. Due to WHO definitions a person who has destroyed his heart by excessive use of alcohol can be included in Cardiomyopthy trials but a person with DMD cannot be.
I find this very disturbing.
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Too right! So do I. Once upon a time, didn't medicos nickname pneumonia the invalid's "friend"? Do you think they still perceive that a crook ticker is also our "friend" in that same sense? I would like to read the source - can you send the link? Thanks.
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You can search for cardiomyopthy at http://apps.who.int/classifications/apps/icd/icd10online/
You get a list of all cardiomyopathy, at index 142.
There is no mention of Dystrophic Cardiomyopathy. Which essentially means that this cannot be a target group for a approved study and Dystrophic Cardiomyopathy will run into this being classified as 2 different ailments.
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