Researchers in the United States have started a revolutionary clinical trial that uses stem cells to treat Thalassaemia in babies while they are still in the womb. The procedure has already been successfully performed on a critically ill second-trimester foetus, resulting in the child being born healthy.
Thalassaemia is the most common inherited blood disorder in the world, with about 5% of the world’s population carrying the gene responsible for it. It causes the body to produce less oxygen-carrying protein (haemoglobin) and fewer red blood cells. Sufferers commonly experience fatigue, weakness, anaemia, and shortness of breath. Some variants of the condition can be quite dangerous, particularly in unborn children.
One of the first patients to be treated using this procedure was born at the UCSF Benioff Children’s Hospital San Francisco in February of this year. The child had received the stem cell treatment four months earlier.
Doctors first realised the child was suffering from alpha thalassaemia major (the most dangerous form of thalassaemia) when they detected life-threatening swelling during an ultrasound. The swelling was a result of the condition starving the body of oxygen. It had caused the foetus’s organs to swell, putting its life in danger.
Doctors immediately began giving the foetus intrauterine blood transfusions to reduce swelling. Once the swelling had subsided, the research team added some stem cells to one of the transfusions.
They used hematopoietic stem cells (HSCs) extracted from the mother’s bone marrow. The HSCs were injected into the umbilical cord of the foetus. Researchers hoped they would move into the foetus’s body and generate healthy red blood cells.
Because a foetus has an underdeveloped immune system, it will readily accept a mother’s stem cells and does not require any immunosuppressant drugs.
Researchers will soon begin testing the procedure on beta thalassaemia, which is the more common form of the disease and sickle cell anaemia.
Source: In Utero Stem Cell Transplants Could Revolutionize Thalassemia Treatment
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