Researchers from the Department of Genetics at the University of Leicester may have found a better way to use stem cells to combat congenital brittle bone disease. The research involved transplanting stem cells into unborn babies which have “brittle-bone disease”, osteogenesis imperfecta (OI).

The disease can cause babies to be born with fragile bones that break very easily. Severe forms of the disease can lead to serious complications including respiratory or heart failure, hearing loss, spinal cord and brain stem problems, and permanent deformities. Babies born with the most severe form of the disease can die shortly after birth.

The disease is caused by a genetic defect that interferes with the production of type 1 collagen, a protein essential for the creation of bone. The defective gene is usually inherited from parents, but it can also spontaneously occur from a genetic mutation.

The €6.6 million study was organised by the Karolinska Institutet and involves several leading European research centres.

Research on stem cells’ regenerative capabilities has increased in recent years with the discovery that some multipotent stem cells can be prompted to form cartilage, bone, tendons and other tissue. 

Researchers at the Karolinska Institutet addressed the shortage of collagen by  producing a strain of stem cell that produces more collagen and strengthens the bone. This stem cell procedure has been tested previously on a small number of children after they were born. It had very positive results — one child is 13 and performing much better than expected. 

However, the treatment has never been given to children at such an early stage in their development.  Scientists will also give repeated doses to children as they grow older.

Thirty babies will be included in the study, with half receiving the stem cell treatment while they are still foetuses and the others receiving their first treatment after birth. They will then receive treatments every 6-months for the first two years. 

After a few years, researchers will then examine the babies to see how many bone fractures have occurred and compare the results with children who did not receive the stem cell treatment.

Research on stem cells has been rapidly advancing in the past decade. If this research project is successful, it will be the latest in a very long list of exciting new stem cell treatments.

Source: Stem cell research to help combat congenital brittle bone disease

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