Clinical proof of this principle has existed for a decade, as Duke physicians have used cord blood to correct heart, brain and liver defects in children with rare metabolic diseases. But until now they lacked the molecular evidence to prove that cord blood stem cells were the root of a cure.

Now, the Duke team has dissected heart tissue to confirm the presence of donor stem cells in heart tissue. Moreover, they showed that donor stem cells had differentiated into heart muscle cells called myocytes, which then produced the critical enzyme needed to halt the progressive heart damage, said Kirsten Crapnell, Ph.D., a research fellow at Duke.

Crapnell will present the team's findings at the International Association of Bone Marrow Transplantation Research meeting Feb. 12-17 in Orlando, Fla.

"We've had convincing clinical evidence that stem cells from umbilical cord blood extended much farther than the blood-forming and immune systems, and that they can differentiate themselves into brain, heart, liver and bone cells," said Joanne Kurtzberg, M.D., director of the Duke Pediatric Bone Marrow and Stem Cell Transplant Program. "But now we have examined heart tissue on a cellular level and proven that donor cells are not only present in heart tissue, but they have become heart muscle cells."

To validate the stem cells' activities, Crapnell dissected and analyzed heart tissue from a 4-year-old boy whose transplant was successful, but who later died of an infection before his immune system was strong enough to fight it. The boy had suffered from a rare metabolic disease called Sanfilippo Syndrome B, in which the body is missing a critical enzyme needed to break down c
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Contact: Becky Levine
levin005@mc.duke.edu
919-684-4148
Duke University Medical Center
16-Feb-2004