PRESS RELEASE: FSHD GLOBAL FUNDS WORLD-FIRST SCIENTIFIC BREAKTHROUGH
Research funded by the FSHD Global Research Foundation has achieved a world-first scientific breakthrough by producing human skeletal muscle from stem cells â€“ a development that is expected to aid in the treatment of FSHD and other muscular dystrophies.
Scientists at Genea Biocells made the discovery of a technique to turn human embryonic stem (hES) cells into skeletal muscle cells in a consistent, scalable and highly efficient manner â€“ which should give researchers an invaluable tool to better understand the mechanisms of muscle diseases. This technique is expected to lead to the discovery and development of effective treatments for muscular dystrophies, a diverse group of debilitating genetic diseases.
â€œThe FSHD Global Research Foundation is committed to finding a cure or treatment for FSHD and this discovery may take us one step closer to that goal. It is critical that our research foundation continues to raise money and raise awareness about the disease so that we can keep funding world leading researchers like Genea,â€ said Chairman and Founder of FSHD Global Research Foundation, Bill Moss.
â€œIâ€™d like to congratulate Genea on the discovery and thank all of our supporters around the world for helping to make this achievement possible.â€
Muscular dystrophies are a diverse group of genetic disorders, characterised by weakness and atrophy of various voluntary muscles in the body.
Facioscapulohumeral Dystrophy (FSHD) is one of the most common forms of muscular dystrophy and is thought to affect seven in every 100,000 people. It is a progressive disorder which can rob sufferers of the ability to smile, talk and eventually walk. Little is known about the cause of the disease and no treatment or cure is currently available.
â€œThis is the first time in Australia that differentiation of human skeletal muscle from pluripotent stem cells has been achieved and the first time in the world that it has been done with significant yields and without cell sorting or genetic manipulation,â€ said General Manager of Genea Biocells, Dr Uli Schmidt.
â€œOur muscle project team, led by Senior Scientist Dr Leslie Caron, undertook a technically challenging task that had never been successfully achieved on this scale before - turning hES cells into the skeletal muscles that allow us to move and smile.
â€œThis world first breakthrough, developed with the support of Australian not-for-profit organisation FSHD Global Research Foundation, means we can produce disease affected human skeletal muscle cells with broad applications in modelling muscular diseases and therefore developing treatments."
â€œThe real world application of this discovery will be human myoblasts, the immediate precursors to skeletal muscle cells, supplied as strictly quality controlled frozen stocks for biotechnology and pharmaceutical companies to use in drug discovery for FSHD. Put simply, this discovery provides a tool which will speed up drug development,â€ concluded Dr Schmidt.
Before the launch of FSHD Global Research Foundation in 2007, there had been no focused research or funding for FSHD research in Australia and very little internationally, leaving research into FSHD 20 years behind other forms of muscular dystrophy.
Since then, The Foundation has received applications for funding from 55 cities around the world and has funded and committed over $3.5 million worth of medical research for twenty two research projects in the USA, Netherlands, France, Italy, Belgium, Canada and Australia, offering hope to thousands of FSHD sufferers and their families around the world.
Thank you for your valuable support,
Chief Executive Officer
FSHD Global Research Foundation