‘Brittle-Bone Babies’ Treated In The Womb With Stem Cells
Researchers have treated babies with congenital bone disease by injecting them with bone-forming stem cells while they were still in the womb.
AsianScientist (Jan. 2, 2014) - An international team of researchers from Sweden, Singapore and Taiwan have treated two 'brittle-bone babies' by injecting bone-forming stem cells into them while they were still in the womb.
The longitudinal results of the treatment are published in the journal Stem Cells Translational Medicine.
Osteogeneis imperfecta (OI) is a congenital bone disease that causes stunted growth and repeated, painful fracturing. Ultrasound scans can reveal fractures already in the fetuses. The babies were treated in utero with mesenchymal stem cells, connective tissue cells that can form and improve bone tissue. The stem cells were extracted from the livers of donors and although they were completely unmatched genetically, there was no rejection and the transplanted cells were accepted as self.
Back in 2005, a paper was published from Karolinska Institutet in Sweden describing how stem cells were given to a female fetus. The present study describes how the girl suffered a large number of fractures and developed scoliosis up to the age of eight, whereupon the researchers decided to give her a fresh stem cell graft from the same donor. For the next two years the girl suffered no new fractures and improved her growth rate. Today she takes dance lessons and participates more in PE at school.
Another unborn baby with OI, a girl from Taiwan, was also given stem cell transplantation by the Karolinska Institutet team and their colleagues from Singapore. The girl subsequently followed a normal and fracture-free growth trajectory until the age of one, when it leveled off. She was given a fresh stem cell treatment and her growth resumed. The girl started to walk and has since not suffered any new fractures. Today she is four years old.
"We believe that the stem cells have helped to relieve the disease since none of the children broke bones for a period following the grafts, and both increased their growth rate," said study leader Dr. Cecilia Götherström, researcher at Karolinska Institutet's Department of Clinical Sciences, Intervention and Technology.
"Today, the children are doing much better than if the transplantations had not been given. OI is a very rare disease and lacks effective treatment, and a combined international effort is needed to examine whether stem cell grafts can alleviate the disease."
Source: Karolinska Institutet; Photo: bradbrundage/Flickr/CC.
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