Researchers have found a new way to save lives of babies in the womb itself by using stem cells
Scientists in Europe are trying to use stem cell therapy in order to cure the brittle bone disease in babies before they are born. This revolutionary technique will involve injecting the stem cells directly into the foetuses in the womb and it will reduce the chances of the baby born with this hereditary problem.
Brittle bone or osteogenesis imperfecta (OI), meaning “imperfectly formed bone” disease is a hereditary ailment that makes the bones of the body so fragile that they break easily. Its presence is known at birth, but it is present only in babies who have a family history of the disease.
Most cases are minor, causing in few bone breaks. Severe forms of the disease can cause spinal cord and brain stem difficulties, and permanent deformities. Also the disease is deadly to babies both before, and soon after birth.
1 The trial
The first official clinical trial of a prenatal stem cell therapy will be inaugurated in Europe next January.
The trial will comprise of foetuses identified with the brittle bone disease. The illness is triggered by a genetic complication with collagen creation and since the bones break easily, it is also very painful. People, who manage to survive the initial onslaught of the disease, may be limited to a wheelchair.
Surgeons around Europe will inject mesenchymal stem cells, which are a type of cell that’s at present is on the route to become bone into foetuses identified with brittle bone disease or osteogenesis imperfecta.
The method is presently in experimental procedure for adult humans suffering from the disorder and many other tissue and immune diseases.
2 How the stem cells were obtained?
The foetal stem cells that will be used in the clinical trials are going to be given by women who have chosen to have an abortion. “A vast majority of women do donate their tissue” said Cecilia Götherström, a senior researcher at the Karolinska Institute and who is also the chief investigator on the clinical trials, “When they have to make such a difficult decision, it feels quite good to help out research” she continued.
This route of study brings the focus on the significance of scientists having constant access to foetal stem cells. “This is just another example where fetal tissue could be really useful and important, not only for biomedical research, but specific patients,” Paul Knoepfler, a stem cell researcher at UC Davis said.
3 The first trial admission group
The trial is controlled by the Karolinska Institute in Sweden and will sign up only 30 pregnant women. Half of the foetuses will be injected with foetal stem cells in the womb, and the process will be repeated again every six months after birth. While the other half will only receive the injections after they are born.
The team at Karolinska has already performed the method on two foetuses that have since been born. The two children have constant access to periodic “boosters” of stem cells, which have significantly lessened the number of breakages when compared with children who suffer from brittle bone disease and receive no stem cell injections.
“It’s important to remember that this is not a cure,” the primary investigator on the clinical trials, Cecilia Götherström of the Karolinska Institute, told Local news. Instead, researchers are hoping to turn severe brittle bone into a mild illness, improving quality of life.
Bone is continuously damaged and reconstructed throughout a person’s life, but the injected stem cells don’t stay in the body. That’s why the clinical trial has to include new stem cell injections every six months for two years.
4 Risks to the trial
As it is with every clinical trial, this trial also has its share of risks involved in the technique used by the researchers.
The stem cells are injected directly into the bloodstream of the foetus, and the said injection has a miscarriage rate of 1 to 2%. Because of the possibility of the cells going anywhere in the body, it is quite possible that they can form a tumor, if they receive a signal and mix with a different kind of tissue. Another major risk is of the foetus’s body rejecting the foreign cells.
“Stem cells are, in a sense, a living drug product,” Knoepfler told local news. “We don’t necessarily know what those cells will do down the road. That has to be balanced against the potential benefits.”
That is the main reason why the first clinical trial is so important. The results of this trial will tell the scientists about how the foetuses will react to stem cell therapy.