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Oxford Neuromuscular Centre

In January 2019, we partnered with the University of Oxford to form the MDUK Oxford Neuromuscular Centre to drive forward the development of new therapies and treatments and increase the capacity of clinical trials for muscle wasting and weakening conditions in the UK. Since then, we‘ve invested over £1.2 million into the Centre.

The Oxford Neuromuscular Centre brings together researchers and experts from across existing sites in Oxford and further afield, with the goal of making Oxford the third hub for muscular dystrophy research in the UK − along with London and Newcastle. Professor Matthew Wood is the Director, alongside Professor Kevin Talbot and Professor Dame Kay Davies as co-Directors,

The centre is making significant progress on the goals and milestones it set out to achieve.

Significant growth in Oxford’s clinical trial capacity

Before the centre was set up, almost no clinical trials for muscle wasting and weakening conditions took place in Oxford due to a lack of expertise and infrastructure, including space, equipment, and other things that facilitate clinical trials, amongst other challenges. Since our investment, and thanks to the recruitment of Professor Laurent Servais, Professor of Paediatric Neuromuscular Diseases, there’s been a significant growth in Oxford’s clinical trial capacity. Today, over 20 clinical trials are either in progress or being set up. These include both interventional, checking if a drug works, and observational, natural history studies, trials.

Dedicated space for new clinical trials for children

In 2019, there was an urgent need for dedicated clinical research facilities where clinical trials for children could safely take place. Thanks to the local NHS Trust and the centre this has changed, and facilities are now available. Even better, the number of these is likely to increase over the coming years, helping to further build the capacity and ability to carry out clinical trials in children with muscle wasting and weakening conditions, as well as other childhood conditions.

A state-of-the-art newborn screening programme

Professor Laurent Servais has set up a state-of-the-art newborn screening programme for spinal muscular atrophy (SMA), with a pilot ongoing in Thames Valley. The newborn screening programme aims to screen a minimum of 10,000 newborn babies for SMA each year over three years. This work also extends well beyond Oxford, with several regions involved in the pilot study. This will provide evidence in support of a UK national screening programme for treatable muscle wasting conditions.

Every baby in the UK is offered screening tests for various conditions in their first week. Currently, SMA is not one of the conditions screened for, despite being included in many other countries’ newborn health checks. While most babies are healthy, for babies who do have a health problem, the benefits of screening can be life-saving. This is the case with SMA – a condition that a baby is born with every five days in the UK.

There are treatments for SMA currently available. It is vital to start treatment as soon as possible upon diagnosis, before symptoms first appear. Once a child starts to show symptoms of SMA, there is already irreversible damage to the nervous system, which affects muscles and movement. However, if treated on time, babies have a great chance of growing up and living long and healthy lives.

“This study represents a unique platform in the UK that we hope will change clinical care for children with SMA now that there are three possible treatments for the condition either recommended by NICE for use by the NHS or in the process of being appraised. Setting the standard of early detection and management of SMA will be a good example for other conditions, such as Duchenne muscular dystrophy’’ Professor Laurent Servais

New therapies in development

The centre is developing new therapies and treatments for several muscle wasting and weakening conditions.

Molecular Patches

One of the most significant achievements of the centre is the development of a drug from ‘bench-to-bedside’ – going from basic research all the way to clinical testing. Working with Wave Life Sciences, Professor Wood and his team have developed a new antisense oligonucleotide (or molecular patch) called WVE-N531. This is a potential treatment for people living with Duchenne muscular dystrophy (DMD) and is currently being tested in an early phase clinical trial by Professor Servais. The potential treatment works by skipping over genetic changes in exon 53 that cause DMD.

More molecular patch therapies for DMD and myotonic dystrophy type 1 (DM1) are also in development with a pharmaceutical company.

Other treatments for Duchenne Muscular Dystrophy

Professors Kay Davies and Angela Russell are developing new treatments to help increase the production of utrophin in people with DMD. Utrophin can replace the function of dystrophin, which isn’t being produced in people with DMD.

Treatments for spinobulbar muscular atrophy

Professor Carlo Rinaldi and his team are working on developing a new gene therapy to treat spinobulbar muscular atrophy (SBMA).

We’ve already made great progress.

But there is still so much that needs to be done. Together, we can change the future of muscle wasting conditions. Join us. Today.