Australasian Science: Australia's authority on science since 1938

Stem Cells Boost Muscular Dystrophy Prospects

By Stephen Luntz

Australian company Genea Biocells has achieved a world-first by turning human embryonic stem cells into skeletal muscle cells in an efficient and scalable manner.

“This 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,” said Genea General Manager Dr Uli Schmidt.

While stem cells have been turned into neurons and cells for certain organs for some time, Schmidt says muscles have proven more complicated, although it is unclear why. Between 70–90% of the cells produced by Genea’s process are functioning muscle cells, making a powerful research tool to model muscular dystrophy.

The research was funded through a Bill Moss Fellowship from Facioscapulohumeral Dystrophy (FSHD) Global. FSHD is a form of muscular dystrophy that starts with an inability to smile or talk and eventually spreads to the rest of the body.

Eventually stem cells may be used to replace muscle damaged by injury or genetic disease, but Schmidt says this will require the muscle tissue to be produced to a clinical standard, which is not yet possible. “It would be necessary to eliminate the other 10% or more cells or prove they are harmless,” he says. “You have to demonstrate there are no pluripotent cells present as they can form tumours.”

In the medium term, however, the capacity to make skeletal muscle tissue should be a benefit for research into all forms of muscular dystrophy, of which Galea is studying cell lines for seven types. Until the establishment of FSHD Global, FSHD was badly under-researched compared with other forms of muscular dystrophy, despite affecting seven in every 100,000 people. As a result, knowledge of the disease lags behind comparable conditions, making the arrival of a new tool particularly significant.