Umbilical Cord Blood Stem Cells Cure Babies With Krabbe’s Disease

Bioethics   |   Steven Ertelt   |   May 24, 2005   |   9:00AM   |   WASHINGTON, DC

Umbilical Cord Blood Stem Cells Cure Babies With Krabbe’s Disease Email this article
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by Maria Vitale Gallagher Staff Writer
May 24, 2005

Washington, DC ( — An amazing new discovery using umbilical cord blood could mean new life for newborn babies with a rare genetic disorder. Umbilical cord blood transplants can save the lives of newborns with Krabbe’s disease, helping their brains to develop normally.

The finding is the result of research by a team of scientists at the University of North Carolina at Chapel Hill and Duke University Medical Center.

Babies with Krabbe disease lack an enzyme necessary for normal functioning of the brain and nervous system.

“The infants become irritable, lose all their developmental skills, become deaf and blind, have seizures and die,” said Dr. Maria Luisa Escolar, the study’s lead author. "It is very painful for parents to watch their children deteriorate this rapidly. This study shows that finally there’s a treatment that offers hope."

Most babies with Krabbe’s disease die before reaching the age of two, but the new findings could help to prolong their lives.

The research adds to a growing body of evidence that cord blood can save children suffering from more than 45 diseases, including Hurler syndrome, Adrenoleukodystrophy, metachromatic leukodystrophy, Tay-Sachs disease, and Sandhoff disease.

Each of the infants in the study who received cord blood transplants as newborns is still alive, with the oldest being six-and-a-half years old. Most of the newborns treated before the onset of symptoms showed continuous improvement in their developmental skills and had age-appropriate mental and language skills.

“Time is of the essence in treating the children before symptoms progress and become irreversible,” said Dr. Joanne Kurtzberg, director of Duke’s Pediatric Blood and Marrow Transplant Program. "The diseases may be uncommon, but the cost to the child, their family and to society at large is enormous when one considers the burden of caring for a severely disabled child. It’s simply impossible to put a price on a child’s life."

Currently, newborns are not screened for Krabbe’s disease, but the study indicates that they should be.

Dr. Mel Levine, director of UNC’s Clinical Center for the Study of Development and Learning, said the research team is making headway in uncovering the inborn lesions that impede learning and development in certain children. “Their research has led to miraculous innovative interventions that can serve as a model for preventing the progression of brain damage in vulnerable babies and preschoolers,” Levine said.

Krabbe’s disease occurs in about one in 100,000 births around the globe. Such children suffer from a loss of myelin, a fatty covering that protects the nerve fibers in the brain. As a result, the nerves in the brain cannot function properly.

Results of the study have been published in the New England Journal of Medicine.

Related website:
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