By Dr Ros Quinlivan, University College London

Here at the National Hospital for Neurology and Neurosurgery in London, we are delighted to learn that Action Duchenne has awarded us a grant to be used as a ‘bridging fund’ for the setting up of new clinical trials for adults with Duchenne muscular dystrophy. We have a growing number of adult Duchenne patients attending our multi-disciplinary service, currently in the region of 100 men affected with the condition. Our goal is to provide the best care that we can and to do our upmost to ensure that adults with Duchenne are given the opportunity to participate in clinical trials.

Currently, there are in the region of 21 pipeline drugs in development for Duchenne. To date almost all of the clinical trials have been undertaken in children and use walking measures, such as the 6 minute walk test, as the primary outcome measure. Very recently, we were really excited to be given the opportunity to participate in a ground-breaking trial, but faced the difficulty of not having sufficient resources in place to set up the trial or run it in the initial stages. This is because funding for industry sponsored trials is reimbursed in small amounts each time a patient visits for the trial. As a consequence, it will take at least 12 months before sufficient funds are reimbursed by the company to employ researchers to perform the trial measurements. Thankfully, our staff are so committed that they have been running this study, so far, on a voluntary basis in their own free time. This is clearly not sustainable in the long-term, since they could choose to stop doing this at any time.

Having insufficient staff to setting up and run clinical trials in the early stages is likely to be a problem that arises again in the future. Because of this, we approached Action Duchenne for a small ‘bridging trial set-up fund’ to enable us to fund researchers for a small amount of time so that the trial can continue to run and so that we have the resource to set up similar trials for adults with Duchenne  in the future.

Thank you Action Duchenne!