In Research
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The Action Duchenne website aims to offer a unique forum for sharing information and ideas in the search for a cure and better medical care for Duchenne and Becker and will at all times attempt to ensure postings are accurate, scientifically peer reviewed and not offensive to individuals. Action Duchenne, however, cannot take responsibility for personal opinions, information and the content of links to other websites. It is essential that you always contact your clinician or GP before taking any course of medication, therapy or supplement.
Looking after your heart in Duchenne
The object is to find out whether starting two specific medicines for the heart - before any signs of ‘heart weakness’ are apparent – can protect the heart better than in the longer-term than waiting to start the same treatments until heart function has begun to weaken.
Heart involvement in DMD …?
You may not be aware that heart is mostly composed of muscle for pumping blood. Heart muscle is not quite the same as muscle elsewhere in the body but it is commonly affected by the DMD process. Most boys with DMD develop early signs of heart weakness (cardiomyopathy) from about their mid teens. The fact that the heart is ‘getting into trouble’ progressively occurs silently and patients remain unaware of it for years. Only when the heart cannot support ordinary day-to-day activities do symptoms occur – energy lack, breathlessness, loss of appetite, fluid retention or abnormal heart racing. It is to detect early signs of heart deterioration (long before any symptoms) that all DMD-boys should have simple heart tests (echocardiogram & ECG) from about the age of 10. Once tests show early ‘heart involvement’, daily treatment with two well-tolerated and commonly used medicines (ACE-inhibitors & beta-blockers) help the heart adjust to the DMD-process. Without treatment heart function weakens progressively over subsequent years, whereas with treatment this can be prevented or at least changed for the better.
The ‘Heart Protection Trial’
Most doctors are now convinced that heart medicines really make a difference in DMD once the heart has begun to weaken and so there is no reason to conduct a research trial in DMD-patients already at this ‘heart-involved’ stage. However, nobody really knows whether starting similar medicines earlier than this offers additional ‘heart protection’ or is worth the table-taking involved. That’s where this research comes in - to answer a gap in our medical knowledge and help improve heart treatments for patients with DMD for the future!
Why not just start the treatments anyway..?
There is a growing list of medicines and other treatments, all offering potential benefits to arm & leg muscles, breathing, and/or heart in DMD. If you believed all the theories, you could be on an impressive range of medicines on a daily basis – steroids, ACE-inhibitors, beta-blockers, anti-oxidants to name but a few. On top of this we hope to have ‘oligos’ (oligo-nucleotide substitution therapy) and ‘morpholinos’ (molecules or ‘drugs’ to modify gene expression) as other promising forms of new treatments very soon. No doubt this list will grow further over the next few years. So there is no shortage of ‘treatments’, it’s now a question of how to get the best out of them …..! Bear in mind that each treatment might be best used instead of earlier treatment or in conjunction with it or might even prevent the beneficial action of another treatment. These are in addition, of course, to the possible harmful effects of treatments used either on their own or in combinations. So from the medical standpoint, its time to take stock and check the extent of benefits for the treatments we have and how to deploy them to best advantage. Although quite a number of ‘medical papers’ have described how patients treated in certain ways fared over time, there are virtually no properly scientific trials (‘randomised controlled trials’) of treatments in patients with DMD. There is a real concern therefore that treatment decisions are really being made in an ‘evidence vacuum’ or, put it another way, that we are ‘building on sand’. It would be of real concern if in the future and recommendations were based on untested-theories! Theories, no matter how elegant or who put them forward, are just opinions of what might or might not be true …… until they are tested properly they may just be misleading!
If you think you might be interested in hearing more about this Heart Protection Trial, future blogs will keep you up to date on when and where the trial is starting and how it is progressing ……