Research into potential treatments for muscle-wasting conditions is at an exciting point, with a range of drugs in late stage clinical trial.
One drug for Duchenne muscular dystrophy, Translarna, is already available in a number of European countries: proof that innovative new treatments are now moving from the lab and into the clinic as approved drugs.
Are we reaching a bottleneck?
As more and more clinical trials come through, the Muscle Centres where they are conducted are finding that that they lack the resources – such as staffing numbers and equipment – to keep pace.
At a meeting in Newcastle with scientists, healthcare professionals, pharmaceutical companies and patient groups, we were alarmed to hear from researchers that UK Muscle Centres are turning down clinical trials due to a lack of capacity
This meeting led to the development of the Newcastle Plan: a joint piece of work with charities to ensure that no new clinical trial is rejected due to a lack of resources. We’re pleased to be working with Action Duchenne, Alex’s Wish, Duchenne Children’s Trust, Duchenne Family Support Group, Duchenne Now, Duchenne Research Fund, Harrison’s Fund and Joining Jack to take this crucial work forward.
What steps are being taken to tackle gaps in capacity?
As part of this work, it was agreed that Muscular Dystrophy UK would lead an extensive audit of clinical trial capacity to find out exactly what centres need, and what they lack, to enable them to take part in clinical trials. This followed an emergency survey conducted by fellow charities, to smaller number of UK sites.
Nearly 20 UK centres took part in our audit, which was chaired by Professor Martin Bobrow and overseen by an Expert Advisory Group of UK and international experts on clinical trial development and of muscle-wasting conditions.
You can read the key findings of the audit here, including:
- Leading Muscle Centres are turning down new trials due to a lack of capacity.
- A lack of acknowledgment of research in clinical job planning means that already overstretched clinical staff are having to carry out research activities in their own time. This is consequently severely limiting centres’ abilities to take part in research.
- Concern from some specialist centres that often onerous testing and sample requirements in early phase clinical trials can be very difficult for young patients to complete, which is affecting levels of patient participation and centres’ ability to deliver results.
- Work on clinical trials is not counting towards specialist training at many centres for medical doctors, physiotherapists and nurses.
With charities involved in the Newcastle Plan, we’re now committed to working to tackling these issues, as well as supporting specialist centres to address staffing and equipment needs in order to develop clinical trial capacity in the UK.
The following experts are members of the Expert Advisory Group that oversaw Muscular Dystrophy UK’s clinical trial capacity audit:
Dr Jonathan Sheffield, Chief Executive, NIHR Clinical Research Network
Professor Dominic Wells, Professor in Translational Medicine, Royal Veterinary College
Professor Rudolf Korinthenberg, Director of Neuropaediatrics and Muscular Disorders at Children’s Hospital, University Hospital Freiburg, Germany
Dr Anna Mayhew, Consultant Research Physiotherapist, MRC Centre for Neuromuscular Disease, Newcastle University
Manoj Thakrar, whose 4 year old son, Shiv, has Duchenne muscular dystrophy
Jeanette Charlton, whose husband, two daughters and grandson have myotonic dystrophy
Christine Medhurst, Senior Investigator Site Development Lead, Pfizer Limited
Edith van Dijkman, Associate Director, Clinical Operations, Biomarin Pharmaceutical Inc
Giles Campion, Head of Neuromuscular Franchise, BioMarin Pharmaceutical Inc
Professor Thomas Voit, Director Designate, Biomedical Research Centre, Great Ormond Street Hospital
Dr Ros Quinlivan, Transition service lead for adolescents and young adults with Neuromuscular Disease at Great Ormond Street Hospital and Queen Square
Dr Helen Roper, Consultant Paediatrician, Birmingham Heartlands Hospital
For more information, please contact Peter Sutton on firstname.lastname@example.org or call 020 7803 4838.