Stem cell study to help fight brittle-bone disease
A pioneering study involving the transplantation of stem cells into foetuses with the brittle-bone disease osteogenesis imperfecta (OI) is to receive funding.
The Horizon 2020-backed, €6.6m ‘Boost brittle bones before birth’, or BOOSTB4, project will be co-ordinated by Karolinska Institutet in Sweden and also sees the participation of the UK’s University of Leicester. OI causes repeated fractures, often before birth, and the study is designed to further test a new treatment on unborn babies.
Since the disease is caused by an inability of the developing body to form normal collagen (a thread-like protein found in bone that has a similar reinforcing function to iron rods in concrete), scientists at Karolinska Institutet have produced a special strain of stem cell which, when injected into the body of sufferers, targets and strengthens the bone by producing collagen. Studies on mice so far have shown a positive response to the treatment, which has also been trialled on children with the disease.
Karolinska researcher Cecilia Götherström, who will also be leading the study, said: “The oldest child to have received the treatment is now 13 and is performing better than expected and is still growing. But we believe that we can improve the treatment for other patients by administering it to the foetus and again in repeated doses during the child’s first years of life.”
Thirty babies will be included in the study, half of whom will receive a stem-cell treatment before birth, and half afterwards. New treatments will then be carried out at six-month intervals over a period of two years to enhance the effect.
The researchers will also assess the results by counting the number of fractures the children in both groups suffer and comparing the results with children who have not been treated with stem cells. The project will start in early 2016.
