The first clinical trial injecting foetal stem cells into babies still in the womb is to start in January. The research will attempt to treat those destined to be born with brittle bone disease, a devastating disease which shortens life expectancy.The trial will be led by Sweden’s Karolinska Institute and in the UK by Great Ormond Street Hospital. Severe brittle bone disease is rare, but can be so serious that women decide to end their pregnancy.The brittleness is usually caused by a defect in the gene that makes collagen, the main building block of bone. As a result, those born with the disease can suffer hundreds of fractures throughout life, becoming confined to wheelchairs with shortened life expectancy. The disease has no cure, and children are normally offered a combination of bone-strengthening drugs, metal rods and physiotherapy are the mainstays of treatment. Sufferers can also have other medical problems from deafness to heart disease.
The trial of 15 unborn babies will see injections in the womb in order to strengthen their bones, allowing them to lead more normal and less painful lives. Until now, stem cells have been tried on two children after birth, but never in the womb. They tolerated the stem cells well, and one, a girl who is now 13, has been able to take up dancing and gymnastics. The new trial involves 30 babies. Half will receive stem cells before birth, and half after, with “top up” jabs for the first two years of life. The researchers will then assess the results by analysing the development of bones and counting the number of fractures that the children in both groups suffer and compare the results with children who have not been treated with stem cells.
Cecilia Götherström, a researcher at Karolinska Institutet’s Department of Clinical Sciences, Intervention and Technology said: “We believe that we can improve the treatment for other patients by administering it to the unborn baby and again in repeated doses during the child’s first years of life.”
The stem cells – “master cells” able to carry out a variety of functions – will home in on the bones and produce the collagen needed to strengthen them. It is hoped that starting the treatment in the womb, before the bones become too damaged, will make it extra-effective.
Professor Lyn Chitty, of Great Ormond Street Hospital, said: “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 one of the first to show that certain conditions can be treated prior to birth, leading to better outcomes for the child.”
Professor Raymond Dalgleish, a Leicester University geneticist involved in the Swedish-led trial, said: “This is an exciting project with the potential to provide much improved quality of life for children affected by severe forms of this disorder.”
Benjamin Jacobs, an expert on the condition and a spokesman for the Royal College of Paediatrics and Child Health, said the jabs were unlikely to cure the disease, but held out hope for an improvement in quality of life.
Cecilia Götherström, a researcher at Karolinska Institutet’s Department of Clinical Sciences, Intervention and Technology said: “We believe that we can improve the treatment for other patients by administering it to the unborn baby and again in repeated doses during the child’s first years of life.”
The stem cells – “master cells” able to carry out a variety of functions – will home in on the bones and produce the collagen needed to strengthen them. It is hoped that starting the treatment in the womb, before the bones become too damaged, will make it extra-effective.
Professor Lyn Chitty, of Great Ormond Street Hospital, said: “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 one of the first to show that certain conditions can be treated prior to birth, leading to better outcomes for the child.”
Professor Raymond Dalgleish, a Leicester University geneticist involved in the Swedish-led trial, said: “This is an exciting project with the potential to provide much improved quality of life for children affected by severe forms of this disorder.”
Benjamin Jacobs, an expert on the condition and a spokesman for the Royal College of Paediatrics and Child Health, said the jabs were unlikely to cure the disease, but held out hope for an improvement in quality of life.
