New research funding brings hope for muscle wasting disease

15 May 2018

Duchenne muscular dystrophy is a severe and complex genetic muscle wasting disease. The condition is diagnosed in early childhood, affecting mainly boys.

Children are often in a wheelchair at the age of 10, and by early adulthood are in need of round-the-clock care and suffer respiratory and cardiac issues. Without effective health provision the average life expectancy is late 20s and currently there is no cure.

Along with the Action Duchenne charity, we are funding research into new stem cell therapies for this condition.

Muscular dystrophies like this are debilitating conditions characterised by progressive weakness and wasting of skeletal muscle. Gene mutations lead to loss of muscle fibres, replacing them with fat and connective tissue.

But muscle stem cells can regenerate muscle fibres and replenish the muscle. This project will explore whether transplanting gene-corrected muscle stem cells generated from patients’ own skin cells can bring new hope for people affected by this devastating disease.

With a combined award of £276,000 (£250,000 from us and £26,000 from Action Duchenne), the research will be led by Dr Yung-Yao Lin, lecturer at the Barts and The London School of Medicine and Dentistry, Queen Mary University of London. This award will fund a feasibility study to produce the data needed to start clinical research.

Dr Lin says: “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.   

Dr Francesca Gliubich, our Director of Grants added: “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.”

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