Health Team

Study: Stem Cell Transplants From Cord Blood Can Help Children With Krabbe Disease

Posted May 19, 2005 2:09 p.m. EDT
Updated November 7, 2006 2:23 p.m. EST

— Researchers at Duke University and the University of North Carolina at Chapel Hill found stem cell transplants from umbilical cord blood helped children with Krabbe Disease. The findings offer hope for babies born with several similar genetic blood disorders. Plus, they point to the need for additional infant screening.

The pediatric stem cell transplant unit at Duke University is filled with stories of despair turned to hope. Many of the children have one of about 45 rare and fatal blood disorders in which they lack key enzymes.

Krabbe disease is one of those disorders. About 5,000 babies in America are born with the fatal disorder each year.

Duke's Dr. Joanne Kurtzberg said that without the needed enzyme, the babies'  brains and nervous systems do not develop normally.

"Eventually, they don't walk or don't talk and (they) develop seizures and blindness and deafness and die by 1 or 2 years of age," she said.

A collaborative study carried out at Duke and UNC Hospitals found cord blood stem cell transplants prevented Krabbe symptoms in young patients.

"A transplant can help treat those diseases because it gives new cells that make the enzyme that the child is missing," Kurtzberg said.

Successful transplants depend on early intervention. Infants born with Krabbe or similar blood disorders must be identified, hopefully before symptoms appear.

Currently, no states perform infant screening for diseases like Krabbe. Duke University is one of only two centers in the country where they could be treated.

"If newborn screening is available, there will be more children identified who need transplants and we will not be able to handle them all," Kurtzberg said.

Kurtzberg said that as more cord blood stem cell transplant centers open, there will be a growing need for publicly funded cord blood banks.

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