Stem cell research has the potential to be one of the greatest advances in biomedical science in human history, giving hope to sufferers of painful, destructive diseases across the world. Although there is a lot more work to be done, just today a group of researchers has announced another major advance in the field. The world’s first in-womb stem cell clinical trial has been announced, which is designed to reduce the severity of a debilitating bone disease very early on in the pregnancy.
Stem cells are known as “undifferentiated,” meaning that they can be coaxed to grow into virtually any type of cell a bioengineer wishes. Broadly, there are two types of stem cells: adult, which are found in a variety of biological tissue types, and embryonic, which are taken from the cell mass within animals that subsequently develops into an embryo. Both can be used to replace destroyed or damaged cells throughout the human body. This means that degenerative neurological diseases including Parkinson’s and Alzheimer’s could perhaps be effectively reversed; autoimmune disorders like multiple sclerosis can also be treated, bringing back physicality to those rendered immobile by it.
Another condition thought to be treatable by stem cell research is osteogenesis imperfecta, known colloquially as brittle bone disease. One in every 25,000 births are affected to some extent by this; babies born with the often-undetected condition can incur fatal fractures, and those that survive live a life fraught with the constantly high risk of suffering disabling fractures.
As reported by BBC News, the trial, beginning next January, will be led and coordinated by both the Karolinska Institute in Sweden and the United Kingdom’s Great Ormond Street Hospital. The international team of researchers hopes that the condition can be treated early on in the development of the fetus.
The disease results from an underproduction of collagen, a structure-forming material found in tendons, ligaments, skin, cartilage and, crucially, bones. The research team wishes to use stem cells that will be taken from the biological material of terminated pregnancies; they will be injected into the fetus in-womb (in utero) as it is developing, and will then hopefully differentiate into healthy collagen-producing cells.
Professor Lyn Chitty of Great Ormond Street Hospital will be one of the researchers carrying out the initial tests on the fetuses to check for the DNA coding errors that lead to the disease. “This is a very serious disease,” she told BBC News. “Our objective is to see if in utero stem cell therapy can ameliorate the condition and the number of fractures.”
This in-womb fetal stem cell procedure has been tested in two cases of brittle bone disease before, but this is the first clinical trial testing how truly effective it is on babies that will otherwise be born with the disability.
Thirty babies will be involved in the pioneering study. The first 15 will be given the stem cell transplant while they are still developing in the womb, and once again after they are born. The other 15 will only have the treatment after they are born. The frequency and intensity of the fractures after birth will be compared to untreated patients born with the disease.
This procedure, if effective, will be another exciting development in a field already at the cutting edge of medical science.
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