Clinical research infrastructure

Early stage research and drug development and are crucial, but without the clinical staff and institutional capacity to administer trials, new treatments will take longer to reach patients.

Great Ormond Street Clinical Trial Co-ordinator

Due to the volume of promising new Duchenne treatments, there is a staffing bottleneck which is delaying treatments getting out of the labs and into clinical trials – to be trialled on boys who so desperately need them. Clinical trials are the best chance for treatment for this generation of boys. The DRF has so far committed to fund a clinical trial co-ordinator at Great Ormond Street Hospital, and hopes to be able to fund further posts nationally in the future, dependent on funds raised in the coming months and years. This is all part of the Newcastle Plan, a collaboration between several UK charities, doctors, regulators and researchers to try to increase staffing for Duchenne clinical trials to help more boys get access to treatments, which has so far funded £1.2 million of posts. Read more about this national collaboration here.

Newcastle University Clinical Research Fellow

We are part of a consortium of six Duchenne charities that has awarded a Fellowship to Dr Michela Guglieri in partnership with Newcastle University and Newcastle University Teaching Hospital to develop and advance the clinical trial agenda for Duchenne muscular dystrophy. The award of £250,000 over five years is being matched by Newcastle University to enable Dr Guglieri to develop the clinical trial capacity for DMD at the John Walton Muscular Dystrophy Research Centre and nationally. Read more about Dr Guglieri’s work and the clinical trial landscape here.

TREAT-NMD programme coordinator

We are also jointly funding Kim Down, a programme coordinator at the John Walton Muscular Dystrophy Research Centre at Newcastle University. Kim works with funders and key stakeholders to ensure the needs and input of the Duchenne community are addressed during the research and clinical process. She is also helping to develop the TREAT-NMD advisory committee for therapeutics.

TREAT-NMD is a network for the neuromuscular field that aims to ensure that the most promising new therapies reach patients as quickly as possible. Since its launch in January 2007 the network has focused on developing tools that enable industry, clinicians and scientists to bring novel therapeutic approaches through preclinical development and into the clinic. It also focuses on establishing best-practice care for neuromuscular patients worldwide. Read more about TREAT-NMD here.