幹細胞 子宮內 治療 brittle bone disease玻璃骨

子宮內嬰注射幹細胞 明年試驗 【經濟日報專訊】英國和瑞典醫學專家宣布，明年1月將在英國進行全球首宗向母體子宮內的嬰兒注射胚胎幹細胞的臨床試驗，歷時兩年。 有助治療「玻璃骨」 試驗由瑞典卡羅琳醫學院（Karolinska Institutet）主導，在英國倫敦大奧蒙德街醫院（Great Ormond Street Hospital）進行，結果有望幫助醫學界治療目前仍無法根治的骨質疏鬆症（俗稱玻璃骨）。 現時全球每2.5萬名出生的嬰兒，便有一人罹患骨質疏鬆病症，出生時有可能會全身多處骨折而夭折；即使嬰兒就算能僥倖生存下來，也要面臨每年多次的骨折、牙齒脆化及聽力下降等問題。 負責為該實驗進行基因檢測的奇蒂（Lyn Chitty）教授指：「這是一個非常嚴重的疾病，我們的目標是驗證在子宮內的幹細胞治療能否改善骨折狀況或病情。」 冀減骨折 強化生長 卡羅琳醫學院專家指，目前學界已進行兩宗針對骨質疏鬆症的子宮內胚胎幹細胞移植，但缺乏臨床試驗，不知道療效如何。 今次是首次在人體內進行試驗，若能減少骨折的頻率或增強骨骼生長，將對學界及產前護理產生巨大影響。

First clinical trial to strengthen babies bones in the womb  Stem cell jabs in the womb could help babies with brittle bone disease and save children a lifetime of pain, researchers say ahead of first trials A pregnant woman is examined as she waits to give birth at a public hospital in Rio de Janeiro  The trial of 15 unborn babies will see injections in the womb, to strengthen bones Photo: AP /Felipe Dan Laura Donnelly By Laura Donnelly, Health Editor2:00PM BST 12 Oct 2015  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."We believe that we can improve the treatment for other patients by administering it (foetal stem cells ) to the unborn baby and again in repeated doses during the child's first years of life" 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. 

This is the first time that stem cells have been used to treat babies in the womb  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. "This is an exciting project with the potential to provide much improved quality of life for children affected by severe forms of this disorder"  Professor Raymond Dalgleish, a Leicester University geneticist 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 the treatment can improve quality of life for those with brittle bone disease 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."