Children with this rare metabolic disease die by the age of 6 because they are missing an important enzyme, alpha-L-iduronidase, which leads to progressive damage in the brain, heart, bones, cartilage, liver and corneas. Stem cells from cord blood provide the needed enzyme in the brain and the rest of the body, thus halting the disease and aiding repair in these organs and tissues, the Duke study showed.
In fact, survival was higher and complications lower among the Duke children who received cord blood than among children previously described in the medical literature who had received adult bone marrow, said Duke's Susan Staba, M.D., lead author of the study. Results of the study in 20 children with Hurler's Syndrome are published in the May 6, 2004, issue of the New England Journal of Medicine. The research was partially funded by the National Heart, Lung, and Blood Institute.
Bone marrow from adult donors can save some Hurler's patients, but an exact match cannot be found in time for more than 50 percent of children, and time is critical in treating these children, said the researchers. Moreover, adult bone marrow fails to engraft in 28 to 37 percent of Hurler's patients, meaning the donor bone marrow does not take hold and begin to grow in the patient.
As an alternative treatment, Duke physician Joanne Kurtzberg, M.D., director of the Pediatric Blood and Marrow Transplant Program at Duke and senior author of the paper, tested the ability of cord blood stem cells to provide the missing enzyme and thereby halt the progressive organ deterioration in these children. Stem cells in cord blood a
Contact: Becky Levine
Duke University Medical Center