A pioneering drug for patients with muscular dystrophy has been approved for use in the NHS, as the centre which helped develop it celebrates 10 years of research and improving patient care.
The John Walton Muscular Dystrophy Research Centre – a partnership between Newcastle University and Newcastle Hospitals – is a world-leader in rare diseases.
Vamorolone, a drug for Duchenne muscular dystrophy (DMD), a paediatric muscle wasting disease mainly affecting boys, has this week been approved by the National Institute for Health and Care Excellence for use in the NHS in England, Wales and Northern Ireland in patients 4 years and older.
The treatment was developed with the help of experts at the John Walton Muscular Dystrophy Research Centre, based in the Centre for Life, and the news is testament to the outstanding work carried out there.
Medical breakthroughs
Bringing together more than 100 employees from Newcastle University and the NHS, the centre’s clinical research and care improves the lives and outcomes of those with all types of rare neuromuscular diseases.
Professor Volker Straub, director of the John Walton Muscular Dystrophy Research Centre, said: “I am very proud that we are celebrating the 10-year anniversary of the research centre.
“Our team is from a variety of backgrounds and works on a comprehensive programme of patient-centred clinical and research activities to further advance understanding of rare diseases, focussing on neuromuscular problems.
“We have been a key part of several medical breakthroughs over the last decade, such as the development of transformative drugs for spinal muscular atrophy, and the starting of UK-first trials of gene therapy for Duchenne muscular dystrophy.
“I’m excited to see what further scientific advances we can continue to make over the next 10-years.”
One of the significant achievements at the centre, has been the development of the new drug, vamorolone, for the treatment of DMD.
Led by Professor Michela Guglieri, the centre was awarded an EU Horizon 2020 grant for £6 million to develop vamorolone as a safe and effective therapy for all patients living with the condition.
The oral drug is a type of corticosteroid and delivers a significant improvement on current treatments and standard of care as it can have fewer side effects, whilst still being as effective.
Newcastle was the leading site involved in studies of vamorolone and it has been one of the biggest treatment advances for DMD in recent years.
Professor Guglieri, professor of neuromuscular disorders at the John Walton Muscular Dystrophy Research Centre and honorary consultant clinical geneticist at Newcastle Hospitals, said: “I am pleased about the positive opinion from the NHS.
“This offers patients with DMD a new treatment option to slow down the progression of the disease while reducing some of the side effects of traditional steroids that affect patient’s quality of life.”
Currently, the centre is also using the power of artificial intelligence (AI) to analyse MRI scans to recognise patterns which identify changes in muscles and speed up disease diagnosis.
The project, called Myo-Guide, is led by Professor Jordi Díaz-Manera and is revolutionising the way neuromuscular patients are diagnosed and assessed, supporting research, and helping clinicians make vital decisions together with their patients.
Sir Jim Mackey, chief executive at Newcastle Hospitals, said:
“The John Walton Muscular Dystrophy Research Centre has been at the forefront of neuromuscular research and scientific breakthroughs over the last decade.
“It brings us great pride to see the centre is leading the way in clinical trials to find much-needed treatments for neuromuscular conditions, which we know will have a significant impact on patients and their families.”
Passion to further medical research
The specialist centre is one of four nationally commissioned for the diagnosis of genetic neuromuscular diseases.
It is named after Lord John Walton of Detchant, a highly respected and prominent neurologist whose life’s work, before he died in 2016 aged 93, has significantly helped patients with muscular dystrophy.
Lord Walton established the North East’s Muscular Dystrophy Laboratories, in which the John Walton Muscular Dystrophy Research Centre has its foundations, which led to Newcastle becoming internationally recognised for its research into the diseases. He headed several medical charities and had key positions in the General Medical Council, Royal Society of Medicine, and the British Medical Association.
His daughter, Ann McNeil, said: “My father was dedicated to his profession for his entire career. From the early days at the Newcastle General Hospital, and the founding of the charity, Muscular Dystrophy Research.
“His work in the House of Lords, and the establishment of the John Walton Muscular Dystrophy Research Centre, was testament to his dedication.
“My father’s passion to further medical research in the field of neuromuscular diseases never left him until his dying day.”
Freddie and Louis’ story
Brothers Freddie and Louis Tanner-Boyer receive care at the John Walton Muscular Dystrophy Research Centre.
The siblings have spinal muscular atrophy (SMA), a rare genetic condition that can cause muscle weakness that progressively gets worse, leading to severe mobility and, at times, breathing issues.
Thanks to the pioneering treatment they have both received at the centre, Freddie, six, and Louis, three, from Gateshead, are doing well and enjoying their lives.
The family had not had any history of SMA until Freddie was diagnosed with the condition as a young child. Sister, Daisy, five, does not have the illness and is also not a carrier of SMA.
Every four-months, Freddie, who requires a wheelchair, is given the ground-breaking treatment, Nusinersen, that has helped stabilise his symptoms.
Louis received gene therapy as a baby and currently has no symptoms of the condition with full mobility.
Mother-of-three, Steph, 33, is a nurse specialist in neuromuscular diseases at the John Walton Muscular Dystrophy Research Centre – a job inspired by the great clinical care of her children.
She said: “We feel so lucky that Freddie and Louis receive care at the centre because the treatment they have both received has been life-changing and we’re very thankful for that.
“The centre is a leader in its field – the expertise of the specialists working there is outstanding and the breakthroughs they continue to make offers hope to patients.”
- Newcastle University and Newcastle Hospitals are both part of Newcastle Health Innovation Partners (NHIP). NHIP is one of eight prestigious Academic Health Science Centres (AHSCs) across the UK, bringing together partners to deliver excellence in research, health education and patient care.
