The paper, authored by Dr Paolo de Coppi (UCL Institute of Child Health and surgeon at Great Ormond Street Hospital) and colleagues in Paris and Padova, showed that damaged muscle tissues treated with cells derived from the fluids which surround the fetus during development leads to satisfactory regeneration and muscle activity. This led to longer survival in mice affected by a muscle variant of spinal muscular atrophy. This is the first time that regeneration of diseased muscle tissue has been obtained using cells derived from amniotic fluid.
The research is published in the journal Stem Cells and represents an impressive development in the growing field of regenerative medicine. Muscle derived stem cells are presently considered the best source for muscle regeneration. However they cannot be used to treat muscular dystrophies as the stem cells themselves are affected in individuals with these conditions. Due to this challenge, other cell sources have been explored but no definitive treatment has been so far successful.
De Coppi’s team has demonstrated that intravenous transplantation of amniotic fluid stem (AFS) cells enhances the muscle strength and improves the survival rate of the affected animals. This is the first study to demonstrate the functional and stable integration of AFS cells into skeletal muscle, highlighting their value as cell source for the treatment of muscular dystrophies.
However, it is still early days in the research, as the work so far has only been carried out in animal models.
Dr Coppi said: "Spinal muscular atrophy is a genetic disease affecting one in 6,000 births. It is currently incurable and in its most severe form children with the condition may not survive long into childhood. Children with a less severe form face the prospect of progressive muscle wasting, loss of mobility and motor function. There is an urgent need for improved treatments.
"We are excited by this potential new approach for regenerating skeletal muscle tissue, but much more research is needed. We now need to perform more in-depth studies with human AFS cells in mouse models to see if using cells derived from the amniotic fluid could be a viable treatment for diseases affecting skeletal muscle tissue."
The study published in Stem Cells was funded by Great Ormond Street Hospital Children’s Charity, Hospital Neker, Paris; and Cariparo and Citta' della Speranza in Italy.
For more information about the work carried out in the UCL Centre for Stem Cells and Regenerative Medicine, visit www.ucl.ac.uk/stemcells
Notes to Editors