For the first time, fetuses in the womb with brittle bone disease will be treated with stem cells.
The goal is that the cells, which can work in many tissues, will
alleviate the symptoms of brittle bone disease, which is incurable.
The rare condition — which affects 1 in every 25,000 births — results
from a genetic mutation and causes severe pain and broken bones.
Those afflicted often have additional medical ailments,
including bone fractures and hearing loss.
This trial will begin with the new year in the United Kingdom and Sweden.
Physicians in Sweden's Karolinka Institute and those at Great Ormond
Street Hospital, a children's hospital in London, will use stem cells
from terminated pregnancies.
"This project may be one of the first to show that certain conditions
can begin to be treated prior to birth, leading to better outcomes for
the child," according to Lyn Chitty of the Great Ormond Street Hospital
in London.
In the trials, 15 babies will be treated with stem cells in utero and
again when they are born. Another group of 15 will be treated after
birth.
The health problems of both groups of babies will be chronicled and
compared with those who have the same disease and were not treated.
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