UK Wedding News
12/10/2015
The project is a collaboration between teams at Great Ormond Street Hospital (GOSH), the UCL Institute of Child Health (ICH), and their colleagues in Europe.
Through carrying out genetic screening in expectant mothers, whose baby is suspected of having severe osteogenesis imperfecta (OI), babies identified with the condition may be treated with a stem cell that increases the production of collagen in the body. This reduces the number of painful bone fractures a child experiences once born.
OI affects around one in 15 people in the UK, and is characterised by painful fractures in bones throughout the body. The condition can also lead to a range of other medical problems, such as discoloured 'brittle' teeth and hearing impairment as a young adult. In a majority of cases, OI is caused by a defect in a gene that produces collagen – a protein that is vital to the strength of bone and other tissues in the body. Currently, there is no cure for the condition and treatments focus on alleviating symptoms rather than treating the underlying cause.
Now, however, a team at the Karolinska Institute in Sweden have developed a special strain of stem cells which, when injected into the body of affected individuals, targets and strengthens the bone by producing collagen. Preliminary studies in mice have shown that this can reduce the number of fractures experienced, as well as some success in alleviating symptoms in a small number of children in their early teens.
A European project to trial the administration of the treatment to babies prior to birth, and in the years immediately following, is now to take place. The trials will start in January 2016 and are set to recruit over a period of two years. It will be coordinated by Karolinska Institutet.
Success of the project will be assessed by comparing fracture numbers in children who had the treatment early in life with those who haven't received injections. If families elect to take part in the trial, a team at University College London Hospital (UCLH) will administer the stem cell therapy in the womb. After birth, the babies will be followed up by the team at GOSH and given injections every six months for two years.
Dr Catherine DeVile, Lead for the Osteogenesis Imperfecta Service at GOSH, said: "OI is a condition that can be debilitating, causing multiple fractures, bone deformity, pain and impaired mobility if severe.
"While injecting these stem cells during teenage years appears to offer some relief in a small number of patients tested, intervention at an early stage in life may lead to more effective results for patients in the long term."
Professor Lyn Chitty, from the genetics laboratory at GOSH, added: "Developments in technology mean that more and more conditions can be diagnosed while a child is still in the womb. If successful, this project may be the one of the first to show that certain conditions can begin to be treated prior to birth, leading to better outcomes for the child."
(JP/CD)
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Project To Use Stem Cells To Treat Bone Disease In The Womb Is Launched
A trial that uses stem cell injections to treat osteogenesis imperfecta, or brittle bone disease, prior to and just after birth, has been launched.The project is a collaboration between teams at Great Ormond Street Hospital (GOSH), the UCL Institute of Child Health (ICH), and their colleagues in Europe.
Through carrying out genetic screening in expectant mothers, whose baby is suspected of having severe osteogenesis imperfecta (OI), babies identified with the condition may be treated with a stem cell that increases the production of collagen in the body. This reduces the number of painful bone fractures a child experiences once born.
OI affects around one in 15 people in the UK, and is characterised by painful fractures in bones throughout the body. The condition can also lead to a range of other medical problems, such as discoloured 'brittle' teeth and hearing impairment as a young adult. In a majority of cases, OI is caused by a defect in a gene that produces collagen – a protein that is vital to the strength of bone and other tissues in the body. Currently, there is no cure for the condition and treatments focus on alleviating symptoms rather than treating the underlying cause.
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Now, however, a team at the Karolinska Institute in Sweden have developed a special strain of stem cells which, when injected into the body of affected individuals, targets and strengthens the bone by producing collagen. Preliminary studies in mice have shown that this can reduce the number of fractures experienced, as well as some success in alleviating symptoms in a small number of children in their early teens.
A European project to trial the administration of the treatment to babies prior to birth, and in the years immediately following, is now to take place. The trials will start in January 2016 and are set to recruit over a period of two years. It will be coordinated by Karolinska Institutet.
Success of the project will be assessed by comparing fracture numbers in children who had the treatment early in life with those who haven't received injections. If families elect to take part in the trial, a team at University College London Hospital (UCLH) will administer the stem cell therapy in the womb. After birth, the babies will be followed up by the team at GOSH and given injections every six months for two years.
Dr Catherine DeVile, Lead for the Osteogenesis Imperfecta Service at GOSH, said: "OI is a condition that can be debilitating, causing multiple fractures, bone deformity, pain and impaired mobility if severe.
"While injecting these stem cells during teenage years appears to offer some relief in a small number of patients tested, intervention at an early stage in life may lead to more effective results for patients in the long term."
Professor Lyn Chitty, from the genetics laboratory at GOSH, added: "Developments in technology mean that more and more conditions can be diagnosed while a child is still in the womb. If successful, this project may be the one of the first to show that certain conditions can begin to be treated prior to birth, leading to better outcomes for the child."
(JP/CD)
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20/03/2020
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