Access to treatments

Duchenne UK launches the DMD Clinical Research Hub

November 2016

Duchenne UK is delighted to announce that Emma Heslop has been appointed as Hub Manager for the Duchenne Muscular Dystrophy Clinical Research Hub.

The mission of the Hub is to ensure that all patients with DMD, including children and adults, have access to clinical research opportunities.

The aim of the Hub is to develop existing infrastructure for DMD clinical trials, so that more sites can run them

The Hub, based in Newcastle, will be a centre for resources, expertise, training and facilitation, for clinical trials in DMD. It will train existing sites to make then ready to accept clinical trials and liaise between industry and sites. 

The idea for the Hub was presented at a workshop in Newcastle in July 2015, organised by Duchenne UK, Joining Jack and TREAT-NMD, to address the lack of capacity to conduct clinical trials in Duchenne clinics in the UK.

How could we facilitate expansion of research capacity in terms of clinicians, research nurses, facilities etc. for DMD trials?

The workshop brought together over 70 clinical, patient organisation, industry and National Institute for Health Research representatives. The Newcastle Plan that resulted has short, medium and long-term strategies to increase clinical trial capacity for DMD in the UK.

  • The one year plan aimed to immediately boost capacity at existing centres of excellence, so that no more trials are turned away. To date Patient Organisations have awarded 1.2 million pounds for posts based in Newcastle, London, Alder Hey and Bristol.
  • A two-year medium term plan aims to build excellence and capacity at existing sites that have trial experience but need extra resource. The DMD hub was an idea that was felt to answer this.
  • The ultimate aim of the five year plan is to ensure that all patients with DMD, including children and adults, have access to clinical research opportunities.

Why do we need The Hub?

Industry can sometimes be reluctant to go to clinical trial sites with no experience and training in running DMD trials, this leads to bottlenecks at existing experienced sites. The Hub’s aim is to make more clinical sites trial ready, but providing training, mentoring and resources.

About our Hub manager Emma Heslop

Emma trained as a biological anthropologist at Durham University and was awarded an MSc (Research) in 2005. She has been part of the muscle team at the John Walton Muscular Dystropy Research Centre at Newcastle University since October 2006 when she joined the TREAT-NMD neuromuscular network of excellence as assistant project manager.

Within TREAT-NMD, Emma was responsible for leading the work relating to ‘Network in Action’ for DMD and SMA and enhancing international collaborations. From 2009 to 2013 she helped form and coordinated the TREAT-NMD Advisory Committee for Therapeutics (TACT) and was subsequently the nominated first point of contact for industry enquiries to the network.

Emma was appointed as RD-Connect Project Manager at Newcastle University in 2013 and with responsibility for leading the strategic development and delivery of the project to ensure the objectives were achieved. 

From January 2017 Emma will lead the development and manage the UK DMD HUB, funded by Duchenne UK.

Emma will be responsible for identifying and recruiting a hub co-ordination team. She will also work with existing trial sites, and with the facilities provided through NIHR and the North Star Network.

Emma says, “I am delighted to take up the post of DMD Hub Manager and excited to launch the DMD Hub. DMD research is at an unprecedented stage in terms of numbers of possible therapies coming to trials. Turning away clinical trials in rare diseases due to lack of capacity is not acceptable. The Hub presents the opportunity to change this situation and we will take this forward in partnership with relevant stakeholders to ensure that all patients with DMD have access to clinical research opportunities in the UK”.

Emily Crossley and Alex Johnson, co-founders of Duchenne UK say: “We are extremely excited about the launch of the Hub and the opportunity it provides to expand clinical trial capacity in the UK. It complements the posts we are supporting through the Newcastle Plan, and support we already provide to the team at the John Walton Muscular Dystrophy Research Centre, where we are funding a DMD coordinator and co-funding a PI to run clinical trials”.

We presented the results of our plan to the European Conference on Rare Diseases and Orphan Products in Edinburgh, May 2016. You can view the poster here 

Published on 24 November 2016

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