鈥淵ou can see during pregnancy that they already have bone fractures in the womb,鈥� says of the Karolinska Institute in Stockholm, Sweden. She is head of what will be the world鈥檚 first ever trial of giving fetuses stem cell therapy in the womb.
The aim is to relieve symptoms of, or even cure, osteogenesis imperfecta, known as brittle bone disease. Babies born with this rare condition have bones that fracture easily, caused by having faulty genes for collagen, the protein that normally reinforces and strengthens bones.
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G枚therstr枚m hopes to prevent this before babies are even born, by injecting them with healthy stem cells that have been extracted from donated tissue from aborted fetuses.
The team will specifically inject mesenchymal stem cells, which should go on to make bone with fully functioning collagen. 鈥淲e hope they will home to bones, reside there and build them up, so they become stronger and grow better,鈥� says G枚therstr枚m.
鈥淲e hope that by intervening so soon, we can treat them before fractures and other damage develops in the womb,鈥� she says.
The team has got approval to carry out 30 treatments. Most of these will be on fetuses, and the stem cells will be injected into their mothers鈥� umbilical veins around 20 weeks into pregnancy. During their first year of life, they will receive four booster injections. A number of babies and infants will also be treated.
The team has already given the treatment to a small number of fetuses, and had promising results. The trial next year will be the first test of the approach to include an untreated control group, to see whether the treatment really does improve the health of babies with this condition.
This article appeared in print under the headline 鈥淒isease fixed before birth鈥�
