22nd May 2020

Duchenne UK funds £228,843 to PhD Post at Newcastle University to work on the data obtained from the FOR DMD study and the impact of corticosteroids on DMD.

For most families, corticosteroids are the main treatment option for boys with DMD: they can help improve muscle strength and function.

However they come with a range of side effects, AND we still do not know which corticosteroid or what treatment regime is best to use. Some boys are on deflazacort, some are on prednisolone. Some are on a daily dose and some take them intermittently.

The reason for these unknowns is that, until now, there had never been a randomised placebo controlled clinical trial to look at which steroids and what regime is most effective.

Well, that’s about to change.

A clinical trial, known as FOR DMD, has been running for 6 years and has now been completed. The trial collected data on 196 boys to better understand the use of different steroid regimes.

The analysis of the main results of this study will be made available later this year.

However, there is a lot more information we can learn about DMD by looking at the data collected in this trial. So, we are funding a PhD researcher, to delve deeper.

The hope is that the information will help provide a wealth of information on the progression of DMD that will help us to better understand the disease. This is important because the better we understand the disease, the better we can design clinical trials and the better their chance of success.

It may also help us in better understanding the effect of other drugs and interventions, including gene therapy.

Dr Michela Guglieri, Clinical Research Fellow, Newcastle University, explained why this is an important project:

The FOR DMD study was developed as an academic collaboration between professor Griggs at Rochester University, US and Professor Bushby at Newcastle University, UK to compare benefits and side effect profile of the three more commonly prescribed corticosteroid regimes in DMD. The study has been run across 5 countries and was designed according to standards of care. The data collected offer a unique opportunity to increase our knowledge about DMD, its progression and the impact of corticosteroids on different aspects of the disease.

A huge amount of data have been collected and the PhD students will help us to make the most out of these data with the principle to share knowledge with clinical experts and the patient community to improve the care of boys and young men with DMD. The analysis of these data will allow us to improve treatment and care, to support clinical trial designs and to describe the natural history of young boys with DMD, which is an essential step to allow us to evaluate the effects of new drugs and interventions, including steroids-alternatives and gene therapy. 

Emily Crossley & Alex Johnson, Co-Founders of Duchenne UK said:

We are very pleased to be partnering with Newcastle on this project. Data, or information collected about patients, is invaluable for advancing care and medical research. Looking at small amounts of data from many patients can help doctors and researchers look for patterns, helping them to improve their understanding and treatment of the disease. In particular we hope this study will give patients greater understanding of the benefits and side effects of different steroid regimes.

What will the PhD researcher do?

They will help ensure that the results from the FOR DMD original study get out to those people who need to know.

They will help create the lay report for the boys (and families) who took part in the study, making sure they understand what they mean.

Crucially, they will help get this message to other patients and the clinical community in general. And it’s not just writing reports – we expect they will present the results personally at conferences and meetings (when the study clinicians are unable to be there).

Next, the researcher will scour the data to see how it can help us address other important clinical and research questions. Of course, until we start looking, we won’t know what else we can glean from the data but we do have some ideas of what we might get: 

  • Knowing the natural progression (history) of the disease in young DMD patients when corticosteroid treatment starts is so important. For example, having that reliable data means we can reduce the number of boys needed for placebo groups in future trials. We will also be able to develop DMD growth charts for use in trials
  • We might better be able to define the best/most reliable outcome measures for new treatments – again, improving the impact of future trials.
  • We know that corticosteroids have some bad side effects – looking in detail at the data may help identify which treatment approaches produce fewer side effects.
  • We know that cardiomyopathy can occur in young DMD boys – looking carefully at the data may give us clues about markers of this aspect of DMD, which we can then try to avoid.

 

Another potential piece of information coming from this study is particularly relevant at the moment. There are several trials underway in which gene therapy is being investigated as a treatment for DMD. Our funding of this research post could provide some very important support information for such gene therapy studies in the future. The current gene therapy trials require stable steroid treatment for 3-6 months before gene therapy begins. A better understanding of the effect of corticosteroids during the first 6-12 months from treatment initiation will be invaluable in helping us see any real additional effect that the gene therapy is having.

 

We would like to thank our partner charities and family funds for supporting this project: Chasing Connor’s Cure, Jacobi's Wish, Team Felix, Help Harry, Archie’s March and Action for Arvin.

If you have any questions, please email [email protected]