Cell therapy for the unborn child

Doctors performed the first successful intrauterine stem cell transplant

Vladimir Mirny, Naked Science

Little Elianna had very little chance of surviving. During an ultrasound, a doctor concerned about the health of her mother, Nichelle Obar, discovered that the woman's fetus suffered from a blood disorder known as alpha-thalassemia. Usually, children with this disease are stillborn or die shortly after birth.

However, despite everything, Elianna now lives at home with her parents in Hawaii. The girl became the first patient in the world to undergo intrauterine stem cell transplantation. Four months after the procedure, Obar gave birth to a healthy-looking girl.

Alpha-thalassemia is a genetic disease that does not allow the blood to produce enough hemoglobin. Without it, red blood cells cannot properly carry oxygen to the cells of the body. Fetuses with alpha-thalassemia may have enlarged organs, swelling, acute anemia and many other life-threatening ailments.

Traditionally, embryos with this disease are given intrauterine blood transfusion by doctors starting from the second trimester of pregnancy. If the therapy works and the child survives, transfusions continue throughout life. There is also an alternative treatment – bone marrow transplantation (doctors transplant stem cells from donor bone marrow) in childhood. This is the only thing that can cure alpha-thalassemia, but such a procedure is very risky and it is also extremely difficult to find a suitable donor.

In Elianna's case, doctors at the Benioff Children's Hospital at the University of California in San Francisco started with a standard intrauterine blood transfusion. However, there was something else in one of the transfusions: blood stem cells extracted from the mother's bone marrow.

"We hope that the cells will reach the bone marrow and help in the production of good red blood cells, which, in turn, will be able to carry oxygen – and thereby cure anemia in the fetus," comments Dr. Tippi Mackenzie, who performed the transplant, in a video about the procedure.

According to Mackenzie, doctors will be able to use stem cell transplants to treat alpha thalassemia, but waiting for delivery only increases the risk of complications.

 "If you perform a stem cell transplant after birth, you will have to give the child a lot of toxic drugs to create a place in the bone marrow. "Since the fetus is more susceptible to its mother's cells than an already born child, doctors can perform a transplant without worrying that the recipient's immune system will reject the treatment."

So far, stem cell transplant treatment confirms its success. Elianna was born at the 37th week of pregnancy - just three weeks ahead of schedule – and weighed slightly less than normal. Everything indicates that the girl is healthy, but it is too early to draw final conclusions about the effectiveness of the technique.

If the treatment worked, in the future it can save many children from chronic illness and even save them from death.

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