Action Duchenne co-funding new research bringing new hope for Duchenne muscular dystrophy

Barts Charity and Action Duchenne have combined forces to support research into new stem cell therapies for Duchenne Muscular Dystrophy. With a combined award of £276,000 (£250,000 from Barts Charity and £26,000 from Action Duchenne) they will be supporting the research of Dr Yung-Yao Lin, lecturer at the Barts and The London School of Medicine and Dentistry, Queen Mary University of London.

“We are delighted to co-fund this project with the Barts Charity. This was an integral part of Action Duchenne’s updated research strategy released last year, supporting promising new techniques that will lead to further advances for Duchenne and Becker Muscular Dystrophy.” 

Dr Lin’s lab has developed novel gene-corrected muscle stem cells, generated from the patient’s own skin, that offer a potential therapeutic agent for Duchenne Muscular Dystrophy (DMD). This award will fund a feasibility study to produce the data needed to start clinical research. Muscle stem cells (myogenic progenitors) can regenerate muscle fibres and replenish the muscle. The project will explore whether transplanting DMD gene-corrected muscle stem cells generated from patients’ own skin cells, will reduce the risk of immunological rejection and the need for life-long immunosuppression, bringing new hope for people affected by this devastating disease.

Muscular dystrophies are debilitating genetic diseases characterised by progressive weakness and wasting of skeletal muscle, which is responsible for voluntary movements and breathing. Gene mutations lead to loss of muscle fibres and their replacement with fat and connective tissue. Current standards of care can delay loss of ambulation, cardiac and respiratory problems, but patients develop progressive weakness leading to immobility. Some muscular dystrophies cause premature death.

Dr Francesca Gliubich, Barts Charity’s Director of Grants said “We are excited at the prospects this research project offers to people affected worldwide.  Muscular dystrophies are genetic disorders with major impact both on life expectancy and quality. At present, there are few effective therapies, and patient-derived muscle stem cells have the potential to provide an effective treatment. This project will provide an essential milestone for progression to clinical trials.”

The Director of Research, Action Duchenne said: “We are delighted to co-fund this project with the Barts Charity. This was an integral part of Action Duchenne’s updated research strategy released last year, supporting promising new techniques that will lead to further advances for Duchenne and Becker Muscular Dystrophy.”

Dr Yung-Yao Lin, Queen Mary University added: “We are grateful to receive the joint award from Action Duchenne and Barts Charity. This award will allow us to further advance the development of cell therapy for treating muscular dystrophies. Our study will pave the way for a first-in-human safety clinical trial. Our collaborative team is very excited to work with both charities on this innovative project.”   

About the organisations

Barts Charity

Barts Charity is a healthcare innovation funder, which aims to provide extraordinary healthcare for 2.5m people in east London, and beyond.

Working alongside the Barts Health NHS Trust’s group of hospitals and Barts and the London School of Medicine and Dentistry (part of Queen Mary University London) we fund world-leading research, state-of-the-art equipment and innovative healthcare projects which go above and beyond what the NHS can provide.

In the year to March 2018, the Charity awarded grants of over £28.5million and at 31 March 2018 there were 135 Barts Charity funded research projects in progress.

Visit bartscharity.org.uk or contact comms@bartscharity.org.uk or 020 7618 1720 for more information.

Action Duchenne

The Action Duchenne charity works tirelessly to fund research specifically for Duchenne and Becker muscular dystrophy. The organisation is the longest running Duchenne-specific charity which also supports and campaigns on behalf of everyone living with Duchenne.

Duchenne is a severe and complex genetic muscle wasting disease. The condition is diagnosed in early childhood and affects mainly boys. Children are often in a wheelchair at the age of 10, and by early adulthood are in need of 24 hour care and suffer respiratory and cardiac issues. Without effective health provision the average life expectancy is in their late 20s. Currently there is no cure.

Contact Neil Bennett, Director of Research neil@actionduchenne.org or 020 7250 8240 for further information

Queen Mary University of London

Queen Mary University of London is one of the UK’s leading universities with 25,332 students representing more than 160 nationalities.

A member of the Russell Group, we work across the humanities and social sciences, medicine and dentistry, and science and engineering, with inspirational teaching directly informed by our research. In the most recent national assessment of the quality of research, we were placed ninth in the UK amongst multi-faculty universities (Research Excellence Framework 2014).

As well as our main site at Mile End – which is home to one of the largest self-contained residential campuses in London – we have campuses at Whitechapel, Charterhouse Square, and West Smithfield dedicated to the study of medicine and dentistry, and a base for legal studies at Lincoln’s Inn Fields.

Queen Mary began life as the People’s Palace, a Victorian philanthropic project designed to bring culture, recreation and education to the people of the East End. We also have roots in Westfield College, one of the first colleges to provide higher education to women; St Bartholomew’s Hospital, one of the first public hospitals in Europe; and The London, one of England’s first medical schools.