Stem Cells and Brittle Bone Disease

Any treatment that is targeted to a foetus tends to raise concerns because the risks and harm caused by an ineffective treatment or one that produces side effects can be frightening. A new stem cell treatment aimed at addressing brittle bone disease – also known as Osteogenica Imperfecta- can however, possibly prolong the lives of children who suffer from this condition, making it an important potential new treatment.

What Is Brittle Bone Disease?

Brittle bone disease is a genetic defect that affects a person’s collagen production. This disruption will eventually lead to brittle, weak bones and stunted growth. There are several types of brittle bone disease – those with type III brittle bone disease usually suffer from fractures whilst still in the womb and rarely live past the early adult years. Even a simple act such as rolling over in bed or coughing can cause a bone to fracture. Clearly, the effects of the disease are devastating and new treatments are crucial to improving the day-to-day health and lives of children with brittle bone disease as well as improving longevity.

International Research

Research into stem cells and brittle bone disease has been carried out in many areas around the globe, including the United Kingdom, Switzerland and the United States. In one study, a team of researchers injected human foetal stem cells through the uterus wall into mouse foetuses that were only fourteen days old. By the age of three months, the mice that were treated had only one third of the long bone fractures that the untreated group of mice had. It’s thought that injections of stem cells, provided they genetically match, can help to improve the production of collagen. Another study used mismatched stem cells from the livers of aborted foetuses.

Treatment was performed on a baby girl who had been identified as suffering from brittle bone disease. Whilst still I the womb, she was injected with stem cells. It was found that by nine months of age, she had not rejected the stem cells, despite concerns she might do so. They also found that she suffered a marked reduction in the number of fractures a child with brittle bone disease typically has – with only three fractures by age two and a half years. Given that children with the disease tend to suffer hundreds of fractures, the results are very promising.

Drugs Versus Stem Cells

There are currently drugs available to treat brittle bone disease but stem cells have added benefits. There is an additional increase in limb length and several children in the United States have, in fact, already received the treatment. All children were treated whilst still in the womb and results thus far are favourable.

Brittle bone disease has a debilitating impact on children and we are fortunate that we can identify those who carry the genetic defect prior to birth. The struggle, however, is to find ways to actually treat the child, given the knowledge that he or she has brittle bone disease. Stem cells offer the potential to improve the lives of children who suffer from the disease by reducing the number of fractures suffered. This means that one day, if a parent hears the painful news that her developing baby has brittle bone disease, there will hopefully be effective options for treatment with stem cells.