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The UK is very poorly prepared to move forward on developing clinical trials and testing new treatments in children and adults with major disabling neuromuscular diseases.
Significant molecular advances have now created opportunities to test new therapies in patients, or in preclinical models, but no systems are in place in the UK to facilitate this process.
Challenges
There is no systematic trials culture for patients with disabling neuromuscular disease. The UK must now move to a 'trials ready' position.
The MRC Centre aims to work with partners to advance clinical trials in neuromuscular disease in the UK.
MRC Centre Programme of activity
We believe that the main current obstacles currently stalling delivery of clinical trials and which will be addressed by the MRC Centre include:
- Lack of infrastructure including issues of trial design, regulatory and biostatistical support.
- Lack of agreed methodology for monitoring disease progression and treatment outcomes.
- Lack of large-scale natural history studies for many neuromuscular diseases.
- Lack of nation-wide patient databases with precise diagnostic criteria.
- Lack of a specific training programme for clinical scientists in neuromuscular disease.
Aims
To overcome the current obstacles to promote a shift to a clinical trials culture in the UK for patients with disabling neuromuscular diseases.
Strategy
The joint London-Newcastle centre addresses each of the obstacles by establishing core activities in clinical trials and in education and training.
The centre has an unrivalled resource of carefully picked patients who are available for studies across all disabling neuromuscular diseases.
We already jointly provide Department of Health funded national services for:
- Limb girdle muscular dystrophy
- Congenital muscular dystrophy
- Muscle channelopathies
- Mitochondrial diseases
Facilities and Expertise
There are state of the art clinical trial facilities available in London and Newcastle.
The clinical trials core group will address the obstacles outlined above and ensure that appropriate support in clinical trials is available to the neuromuscular centre.
The plan of work of the clinical trials core group is likely to result in a sustainable, durable expansion of clinical trials enabling us to take full advantage of these facilities.