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Duchenne muscular dystrophy clinical trial

Drug development project receives £4.6m to help boys with rare illness

Published on: 12 March 2016

Millions of pounds have been invested in a drug development programme to help boys with a rare life-limiting disease.

The John Walton Muscular Dystrophy Research Centre at Newcastle University has received £4.6 million from the European Commission for a pioneering clinical trial into the new drug, vamorolone.

The European project, called VISION-DMD, involves 10 countries and is being led by the North East to help children with Duchenne muscular dystrophy (DMD).

DMD is a genetic muscle wasting illness which affects approximately 50,000 boys worldwide. It causes muscle degeneration and a decrease in life-expectancy.

Cost-effective drug development

Kate Bushby is Professor of Neuromuscular Genetics at the John Walton Muscular Dystrophy Research Centre and honorary consultant geneticist at Newcastle Upon Tyne Hospitals NHS Foundation Trust.

She said: “This project has been designed to ensure a timely and cost-effective drug development for DMD, which is highly demanded by patients, families and regulatory authorities.

“It is great that the John Walton Muscular Dystrophy Research Centre has received this funding and can, once again, help lead the way in groundbreaking research to improve the lives of boys with DMD.”

The European Community Horizon 2020 project has provided funding for the four-year VISION-DMD scheme, which is the first clinical trial of its kind.

It is due to working with the Collaborative International Neuromuscular Research Group, Washington US, and ReveraGen, the company who developed the drug, that this funding has been granted. 

Anti-inflammatory drug

Steroids remain the main long-term treatment for the condition as they effectively reduce inflammation. Vamorolone – also known as VBP15 - is an anti-inflammatory drug.

It is expected that the new treatment will improve muscle strength in patients without the side-effects seen in steroid treatments, such as mood swings, stunted growth and weight gain.

Dr Michela Guglieri is Principal Investigator of the trial at the John Walton Muscular Dystrophy Research Centre and also works at the Newcastle Clinical Research Facility at the Royal Victoria Infirmary.

She said: “We have established steroids as a standard therapy in DMD, but the side-effects often blunt the benefits.

“Patients and families have been waiting for a new drug which can retain the good properties of steroids but prevent or limit their downsides.

“Vamorolone could significantly change the treatment and management of all cases of DMD. It does not represent an alternative, but an addition, to interventions currently in development.

“Our vamorolone programme at the John Walton Muscular Dystrophy Research Centre combines the expertise of our clinicians with highly specialised drug technology.”

The clinical trial comes under the umbrella of Newcastle Academic Health Partners, as it is a collaboration involving Newcastle Upon Tyne Hospitals NHS Foundation Trust and Newcastle University.

100 boys will be on the clinical trial

ECRIN European infrastructure for clinical trials will coordinate the enrolment of approximately 100 boys with DMD in 10 European countries. Trials are expected to begin in 2017.

Emily Crossley, Director of Duchenne Children’s Trust, a foundation co-supporting the VISION-DMD programme, said: “As parents we see the benefit of steroids, but also the side-effects. We would embrace an alternative treatment.”

ReveraGen, a company specialising in therapeutic approaches to muscular dystrophy, has been key to developing vamorolone.

Dr John McCall, medicinal chemist and co-founder of ReveraGen, said: “Steroids are complex drugs with many actions, but we hope that vamorolone will have increased efficacy combined with improved safety for patients.”

Case study

Little Ryan Bennett was diagnosed with Duchenne muscular dystrophy more than a year ago.

The schoolboy, of Bishop Auckland, County Durham, continues to live life to the full as he remains an active five-year-old.

His mum Rebecca Daniel, 29, who also has daughter, Amy Bennett, 10, has welcomed the VISION-DMD project.

The student nurse said: “It’s great that millions of pounds have been given to help improve treatment for those with Duchenne muscular dystrophy.

“This drug development programme offers hope that significant advancements will be made in the years to come to help patients with the condition.

“We keep our fingers crossed that research developments in the future will help Ryan and others like him live as good a life as possible.

“We could not live in a better place than the North East for Ryan as the research that’s carried out at the John Walton Muscular Dystrophy Research Centre is excellent.”

Rebecca Daniel with her son Ryan Bennett

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