“WEDNESDAY, May 18 (HealthDay News) — Umbilical cord blood stem cells can help save infants with the fatal genetic disorder Krabbe disease, researchers report in the May 19 issue of the New England Journal of Medicine.
Investigators found that giving umbilical cord blood stem cells to these infants before they develop symptoms of the disease can preserve brain development and save their lives. Without an immediate transplant of stem cells, infants with Krabbe disease rapidly start to lose all cognitive and motor functions and die by 2 years of age.
This is the first study to demonstrate a lifesaving treatment for children with Krabbe disease. Children with the disease lack an enzyme critical to forming the myelin sheath that protects developing brain cells from damage.
Researchers at Duke University Medical Center and the University of North Carolina at Chapel Hill studied 11 asymptomatic babies with Krabbe disease (aged 12 to 44 days) and 14 symptomatic infants (aged 142 days to 1 year) treated with umbilical cord blood stem cells from unrelated donors.
The stem cells took hold in all the children. All of the asymptomatic infants survived the stem cell transplant, compared to 43 percent of the symptomatic infants.
The researchers noted that while the infant’s age and symptom severity at the time of transplant are major factors in determining outcome, the source of stem cells also plays an important role.
Compared to stem cells from adult bone marrow, umbilical cord stem cells provide a better and quicker correction of enzyme deficiencies. Cord stem cells travel to the brain faster and repair deficiencies in both the central and peripheral nervous systems.
Progression of disease stabilizes two to four months earlier in infants who receive cord stem cells than in those who receive stem cells from adult bone marrow.
The U.S. National Library of Medicine has more about Krabbe disease