Transplantation therapy was tested in patients with infantile Krabbe Disease to determine whether early administration of these cells would provide sufficient enzyme to the peripheral and central nervous systems to facilitate normal myelination.

It has been shown that stem cells from bone marrow and umbilical cord blood can differentiate into non-hematopoietic tissues including brain, bone, liver, pancreas and cartilage. Transplantation of healthy donor cells into children with certain inborn errors of metabolism can halt disease progression and, in some cases, repair damage caused by the disease.

Cord blood is less particular than bone marrow because the stem cells are undeveloped and don't require a perfect match to the patient. The risk of cord blood cells mounting a serious immune attack against host tissues is only about 10%, the same as with a perfectly matched related bone marrow. Treatment with bone marrow or cord blood transplantation requires exposure to 1 - 2 weeks of high dose chemotherapy.

A Brief History of Cord Blood Transplants

The first unrelated cord blood transplant in the United States was performed at Duke University Medical Center in 1993. Since then, cord blood transplants have become increasingly common. Nationwide over 500 cord blood transplants are performed each year, compared to about 6,600 bone marrow transplants. This procedure has saved the lives of more than 1,000 patients worldwide.

Dr. Joanne Kurtzberg is the Director of the Pediatric Bone Marrow and Stem Cell Transplant program at Duke. She is a pioneer in cord blood transplants and helped revolutionize the procedure, which she has been working on for more than 20 years.

"Now that an effective therapy is available for infants with Krabbe Disease, newborn screening is essential to identify affected babies at a time when they can maximally benefit from treatment." - Dr. Joanne Kurtzberg