Major investment paving the way to new clinical trials

Published Date
23/12/2015
Author
Jonathan Kingsley
Category
Care & Support

A major investment of over £1.2 million has been announced today, to fund key roles at three neuromuscular centres of excellence. The commitment has been driven by a number of Duchenne charities and Great Ormond Street Hospital charity collaborating together to boost clinical trial capacity at UK muscle centres. We’re delighted to announce that Muscular Dystrophy UK’s initial contribution is set at £361,000 over the coming two years.

Earlier this year, Muscular Dystrophy UK joined forces with a number of other Duchenne charities as part of the ‘Newcastle plan’ initiative, following concerns from clinicians and patient groups that trials were being turned down due to a lack of capacity and specialists in post.

This increased funding over the next few years, and beyond in some cases, will now help recruit the following key posts:

Three new posts at Alder Hey Children’s Hospital in Liverpool

  • Research Physiotherapist
  • Specialty Doctor
  • Trial Co-ordinator

Nine new posts at Great Ormond Street Hospital in London, with the Great Ormond Street Hospital charity and the GOSH Trust also making a contribution: 

  • 2 Clinical Research Fellows
  • Physiotherapy Assistant
  • 2 Research Physiotherapists
  • Clinical Trial Co-ordinator
  • Data Manager (funded by GOSH Trust)
  • Professor of Neuromuscular post
  • Senior Lecturer

Four new posts at the Newcastle Muscle Centre:

  • Clinical Research Associate
  • Clerical Assistant
  • Research Physiotherapist
  • Clinical Research Nurse

The charities providing funding for these posts include Muscular Dystrophy UK, the GOSH charity, Action Duchenne, Alex’s Wish, Duchenne Children’s Trust, Duchenne Now, Duchenne Research Fund, Harrison’s Fund and Joining Jack. The Duchenne Family Support Group, DMD Pathfinders and Join our Boys Trust backs the ‘Newcastle plan’ and the funding.

Muscular Dystrophy UK’s initial contribution over two years will be £361,000, funding four new roles:

  • A research physiotherapist at the Newcastle Muscle Centre;
  • A Clinical Trial Co-ordinator at Alder Hey – and will also link across to Manchester Children’s Hospital;
  • A Senior Lecturer and a Professor at Great Ormond Street Hospital in London – also be supported by the GOSH charity.

This builds on Muscular Dystrophy UK’s existing commitment in Newcastle and London, funding two Clinical Trial Co-ordinators, as well as the North Star natural history database for Duchenne and SMA.

In addition to working closely with other charities in establishing the need for these posts, we set up an expert group to run a UK-wide audit to investigate clinical trial capacity in the UK.  Chaired by Professor Martin Bobrow, the audit group helped to guide Muscular Dystrophy UK in making this significant investment at these three centres.

Manoj Thakrar, who together with his wife Sejal set up the Smile with Shiv Family Fund after their son Shiv was diagnosed with Duchenne muscular dystrophy, said:

It’s really encouraging that Duchenne charities and families like mine can work towards one clear goal and mission. A significant investment has now been made to improve clinical trial capacity, which will help ensure more trials can start and more patients recruited.  As a member of the clinical trial audit group and from what has been discovered and highlighted in work done in the past by all the charities, I’ve seen the challenges facing many centres, and it’s clear that we need to continue pushing to ensure those neuromuscular specialists have the resources they need.

Commenting on these developments, Nic Bungay, Director of Campaigns, Care and Information at Muscular Dystrophy UK, said:

Today’s announcement of sixteen brand new roles represents a huge step forward and a major boost to clinical trial capacity.  The pipeline for potential clinical trials looks very promising and we must ensure that Muscle Centres have the resources they urgently need.

 

It is also vital that Hospital Trusts, the NIHR and Biomedical Research Centres also play their part, so that funding for these posts remains sustainable in the longer term.

 

We are also keen to help develop further support for all the Muscle Centres involved in clinical trials across the UK and will continue working closely with neuromuscular teams throughout the coming Year.

Professor Kate Bushby, from the John Walton Muscular Dystrophy Research Centre at Newcastle University said:

I am delighted that we have already received such tangible results in such a short space of time and hope that these posts will be the start of building real and sustained trial capacity, and will help to keep the UK at the forefront of exciting trials coming through for Duchenne muscular dystrophy.

Dr William van’t Hoff, National Specialty Lead for Children at the NIHR Clinical Research Network, said:

The National Institute for Health Research (NIHR) Clinical Research Network is delighted to have worked in partnership with patient organisations and hospital sites to quickly facilitate increased clinical support for neuromuscular trials. Building on the success of previous studies and utilising this national collaboration, the UK is well-placed to conduct more studies to improve treatments for children with these disorders and to increase the number of sites able to conduct this important research.

For more information on our work to address clinical trial capacity and to get involved, please contact Peter Sutton on p.sutton@musculardystorphyuk.org or call 020 7803 4838.

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