Babies with Osteogeneis Imperfecta Helped by Stem Cell Grafts

Stem cell grafts may provide a key treatment for children born with osteogenesis imperfecta (OI), a congenital bone disease that causes stunted growth and painful fracturing. Researchers believe that by using mesenchymal stem cells, connective tissues in the infants' bodies can form and improve bone tissue.

"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," lead study author Dr. Cecilia Gotherstrom who is also a researcher at Karolinska Instituet's Department of Clinical Sciences said, via a press release. "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."

Researchers extracted stem cells from the livers of donors. Though these cells were not matched genetically, findings showed that there was no rejection of the transplanted cells to be self-accepted in the children.

The study team identified several children suffering from OI. The first describes a girl discovered in 2005 from Sweden and how stem cells were given to her in the womb. Though she suffered a large number of fractures and developed scoliosis up until the age of eight, researchers decided to give her a fresh stem cell graft from the same donor. Over the next two years, she suffered no new fractures and increased growth rate.

Another girl from Taiwan was also given stem cell transplantation in the womb during the same time period. She followed a normal and fracture-free growth trajectory up until the age of one, at which she was given a new stem cell treatment from the same donor. Her growth resumed with no new fractures.

Lastly, a boy from Canada with the same mutation as the child from Sweden was studied but not given cell therapy. Numerous problems involving fractures and thoratic vertebrae eventually lead to his death 5 months later. His problems harmed breathing, and he died shortly after of pneumonia, according to the study.

Though more research is required to confirm the success of the grafts, researchers are hopeful that injection of bone-forming stem cells in utero can help prevent future fractures and increase growth for children that will suffer from the health condition as they age.

More information regarding the study can be found via the journal Stem Cells Translational Medicine.