Cord Blood Transplant May Treat Metabolic Disorders
Cord-blood transplants to treat genetic metabolic disorders in babies while they’re still in the womb.
The new method uses a small, select number of therapeutic stem cells that have been treated to speed and improve engraftment (acceptance of the transplant by the body), explained Dr. Joanne Kurtzberg, a professor of pediatrics and pathology and director of the pediatric blood and marrow transplant program at Duke University Medical Center in Durham, N.C.
The new procedure in a pilot trial open to pregnant women at risk for having children with fatal metabolic disorders, including Krabbe disease, metachromatic leukodystrophy, Pelizaeus-Maerzbacher disease, Tay-Sachs disease and Sandoff disease.
If untreated, these metabolic disorders can lead to bone, brain and central nervous system problems, and early death.
Cord-blood transplants after birth have proven successful in treating inherited metabolic disorders. Transplant timing is critical, Kurtzberg stressed.
“The idea is to give the baby cord-blood stem cells from a healthy donor that have the potential to provide healthy genes that can replace the ones that aren’t working properly in the baby’s own cells,”.
In general, the earlier the transplant, the more likely it will work. That means that performing the transplant before the baby is born is ideal.
During the procedure, donor cells are injected directly into the fetus’s abdomen at 12 to 14 weeks’ pregnancy. At birth, the baby will be tested to see if the donor cells are present, and if they’re fixing the malfunctioning genes. If not, the baby would be eligible for conventional cord-blood stem cell transplant within a few weeks.
A major advantage of using cord blood as a source of stem cells is that the severity of graft-versus-host disease, a major complication of marrow transplantation, appears to be less, even in mis-matched transplants. Children who have transplants using matched sibling cord blood appear to have less than a 10% chance of developing graft-versus-host disease, significantly less than observed using bone marrow from the same type of donor. The overall incidence of graft-versus-host disease in unrelated transplants, not unexpectedly, is higher; but the incidence of the most severe form also appears to be markedly less than that observed with using marrow from an unrelated marrow donor. Cord blood transplantation is associated with a smaller risk of life-threatening graft-versus-host disease when compared to marrow transplantation.
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