Alex’s Wish help fund study to improve our knowledge of heart care in patients with DMD

12 May 2021

Alex’s Wish has invested £10,000 towards a study funded by Duchenne UK that aims to improve our knowledge of treating heart muscle disease in patients with Duchenne muscular dystrophy (DMD). 

Almost all people with DMD will develop heart muscle disease, or cardiomyopathy, at some stage. There are drugs that are believed to slow down damage and protect the heart. However, no clear data exists for their effective use in DMD.  

In 2011, a clinical trial, called The Heart Protection Study, was set up to see if starting two existing heart medications in combination (perindopril and bisoprolol) could protect against the development of heart muscle weakness in young boys with DMD. The trial included 75 patients – 5-13 years old at entry, and ended in 2018 when all patients had completed three years of follow-up.   

The results did not show that there was any difference between boys on active as compared to dummy treatments in terms of reduction in the damage to heart. However, this negative outcome could have occurred for a number of reasons. Most participants were also taking steroids, which is known to delay the onset of cardiomyopathy by about two years. Also, DMD progresses at different rates, so these young patients may not yet have been affected by cardiomyopathy.   

That’s why Duchenne UK is giving a grant of £68,496 to Dr John Bourke at the Newcastle Upon Tyne NHS Foundation Trust to further study the information from that trial to see if we can get clearer answers about the value of these medications.  

The grant will enable Dr Bourke to follow up on the same patients who were in the original trial, and use data collected from them in the years since the trial ended in 2018. The patients continued the heart medications meaning that there are now an additional 3 years of heart reviews, collected as part of their regular check-ups. This should give us a clearer picture of the impact of these two drugs on heart function.  

The advantages of this study are that there is little inconvenience to patients and their families, with no extra hospital visits; a short study time frame of only 10 months; and all preparatory work in place. Also, if the hoped-for benefits of these two drugs is confirmed, then these medicines are readily available and could be routinely prescribed even earlier in boys with DMD.  

Duchenne UK is very supportive of examining patient data to help us understand more about DMD and to identify potential treatments. We would like to thank Dr John Bourke for bringing this study to our attention and we look forward to sharing the results with you. 

This Project was funded by the fabulous collaboration of the Duchenne Community under Duchenne UK – Alex’s Wish, and the following Family Funds: Jack’s Mission, Help Harry, Archie’s March, Team Dex, Jacobi’s Wish, William’s Fund, Team Oscar, Following Felix, Ralphy’s Fund, Defending William Against DMD, Love for Leon, Henry’s Hurdles, Ben vs Duchenne, Moving Muscles for Marcus.

Posted in Funding, Research News