Stem cells against bone fragility syndrome

An international team of researchers led by Dr. Cecilia Götherström from the Karolinska Institute (Sweden) plans to launch in January 2016 the first of its kind clinical trial in which unborn children with osteogenesis imperfecta will be injected with stem cells.

Osteogenesis imperfecta, or bone fragility syndrome, is a severe hereditary disease in which children suffer from frequent fractures of various bones of the skeleton from birth. To date, the disease is incurable. Quite often this leads to physical disability, posture disorders and stunted growth. Narrowing of the chest in such children can lead to impaired respiratory and pulmonary functions. 

The reason for these disorders is the inability of the developing organism to produce collagen, a fibrous protein that is a kind of "reinforcement" of bone tissue. In search of a solution to this problem, researchers at the Karolinska Institute have created a line of stem cells that, after being injected into the affected body, move into bone tissue and strengthen it through collagen synthesis. Experiments on a mouse model demonstrated a good response to therapy, as well as the introduction of cells to several children with osteogenesis imperfecta. The eldest of these patients is now 13 years old, he continues to grow and demonstrates results that exceed the expectations of specialists.

However, despite the promising results, the researchers believe that the effectiveness of therapy can be significantly improved by introducing stem cells to unborn patients with repeated injections during the first years of life.

In total, 30 children are planned to be included in the clinical study, half of whom will receive the first dose of cells between 20 and 34 weeks of pregnancy, when the embryo's genitals have already formed and there is no danger of reproductive function disorders in the future (and even more so – the formation of germ cells with someone else's genome), and the second half – after birth. Subsequent injections, the purpose of which is to strengthen and consolidate the effect, will be carried out every 6 months for two years. After that, the researchers will summarize the results by analyzing the development of bones and counting the number of fractures in patients of both groups, followed by comparing the data obtained with the corresponding parameters for children who have not undergone stem cell therapy.

Evgeniya Ryabtseva

Portal "Eternal youth" http://vechnayamolodost.ru based on the materials of Karolinska Institutet: Unique stem cell brittle-bone study starts.