Duke was the first to use umbilical cord blood from unrelated
donors to cure life-threatening cancers, rare immune
deficiencies, and metabolic diseases. Because patients do not
require an exact blood “HLA” match with cord blood as they do
with bone marrow, it is easier to find a cord blood match,
providing access to this life-saving therapy for patients unable
to find a fully matched donor. Duke was also the first to prove
that bone marrow transplants could cure severe combined immune
deficiency if administered in the first months of life. Our
researchers showed that transplantation can also be an effective
treatment for Hurler’s syndrome, a rare metabolic disease (MPS1)
in which patients suffer from progressive brain, liver, heart,
bone, cartilage, and cornea damage due to a missing enzyme. In
addition, transplantation has proved effective in treating some
cases of Krabbe disease, Sanfilippo syndrome (MPS III),
metachromatic leukodystrophy (MLD), and adrenoleukodystrophy
(ALD). Duke researchers are continual