Findings published today in Scientific Reports shows for the first time that stem cells gathered from the amniotic fluid of pregnant women can strengthen bones, reduce breaks by 78%, and improve the quality of bone tissue in mice with brittle bone disease.
If these cells show the same positive effect in humans, it could mean significantly fewer fractures, reduced visits to hospital, and less chronic pain, resulting in individuals leading more active and unrestricted lives. Ultimately, the researchers hope that this treatment option will see an end to all symptoms associated with these conditions.
Pascale V Guillot, Senior Lecturer in Stem Cell Therapy at UCL GOS Institute of Child Health, who led the study, says: “Amniotic stem cells are quick, easy and safe to obtain. This is the first time this type of stem cell has been used to successfully strengthen bones and improve their quality. The discovery could have a profound effect on the lives of patients who have fragile bones and could stop a large number of their painful fractures.”
This project is one of over 1000 research projects being carried out at the UCL GOS Institute of Child Health and Great Ormond Street Hospital as part of their research programme, which aims to develop treatments and cures for conditions affecting not only children and adolescents, but global populations across all ages.
Brittle bone disease, also known as osteogenesis imperfecta (OI), affects an average of 70 babies per year in the UK and inflicts painful bone fractures across the body. These fractures start to occur in the womb and continue throughout a person’s lifetime. In contrast, osteoporosis affects over three million people, mainly over the age of 50, in the UK. It therefore represents a significant burden to the NHS, with estimated costs of osteoporosis fractures alone reaching billions of pounds each year. There is currently no cure for either condition and treatments focus on alleviating symptoms.
Standard treatment comes in the form of tablets known as biphosphonates, but they can be detrimental to bone-forming cells and sometimes lead to the cells’ premature death. The use of stem cells to promote bone growth has the potential to be a much better treatment option as they do not not have any adverse side effects.
Pascale V Guillot and colleagues injected newborn mice who had brittle bone disease with human stem cells found in the fluid that surrounds the baby during pregnancy. They then compared their bones to mice with brittle bone disease who had not received the stem cells. They found that amniotic stem cells appeared to protect the fragile bones of the mice by increasing their strength, plasticity, structure and tissue quality. They believe this is because the stem cells encourage the development of osteoblasts – the cells that make bone.
In the case of OI, babies are born with fractures they sustain in the womb. The authors suggest, if the results of this study can be replicated in humans, injecting stem cells at birth or in the womb, would allow these babies to develop healthier skeletons. This is because the transplanted cells would stimulate bone-forming cells to produce more bone that is more mature. As a result, the bones would be stronger and more resistant. They expect this would need to be repeated several times a year to replenish the beneficial cells as bones grow and develop.
The team have now started work to ‘superpower’ these cells, in they hope that they can strengthen their effects even further and eradicate fractures in individuals with fragile bones completely, which is considerable burden for people who are immobilised, the more senior population and astronauts.
The researchers hope to begin a clinical trial to test this procedure in humans in the next 1-2 years.
Counteracting bone fragility with human amniotic mesenchymal stem cells
DOI: 10.1038/srep39656 www.nature.com/articles/srep39656
This work was supported by the National Institute for Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children NHS Foundation Trust and University College London. It was also supported by grants from Action Medical Research, Rosetrees Trust and Newlife Foundation.
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Notes to Editors
Great Ormond Street Hospital for Children NHS Trust is the country’s leading centre for treating sick children, with the widest range of specialists under one roof.
With the UCL Great Ormond Street Institute of Child Health, we are the largest centre for paediatric research outside the US and play a key role in training children’s health specialists for the future.
Great Ormond Street Hospital Children’s Charity needs to raise money to support the hospital to give children who need help the most the best chance for life. The charity funds patient and family support programmes, provides the latest medical equipment and supports the essential redevelopment of the hospital. It has also launched a five-year strategy to support research in some of the most serious and complex childhood diseases. Great Ormond Street Hospital Children’s Charity is the largest dedicated funder of paediatric research in the UK and our work is entirely funded through the generosity of supporter donations. For more information visit www.gosh.org