Can cord blood stem cells correct Inherited Metabolic Disorders?

Answer: Probably. Some hereditary disorders cause the metabolism to gradually destroy the nervous system, as well as damage other organs. One class of hereditary disorders is leukodystrophies, in which the cells sheathing nerves are improperly developed or maintained, and gradually break down. Another class of hereditary disorders is storage disorders, in which cells are damaged by the abnormal accumulation of waste products. There are different types of storage disorders: Mucopolysaccharidoses (MPS) Storage diseases (includes Hurler’s Syndrome, Sanfilippo Syndrome) and Lysosomal Storage diseases (includes Gaucher Disease, Tay-Sachs).

The team lead by Joanne Kurtzberg, M.D., at Duke University, is conducting trials of Cord Blood transplants in very young children with various inherited disorders. So far, they have presented successful results with Sanfilippo Syndrome, Hurler’s Syndrome, and Krabbe Disease. References:

• Duke University press release Jan 2004, also presented at American Society of Hematology Dec 2003 meeting
• Medscape CME on Hurler’s Syndrome, based on
• Staba, SL, et al., N Engl J Med. 2004; 350:1960-1969. “Cord-blood transplants from unrelated donors in patients with Hurler’s syndrome.”
• Escolar ML, et al., N Engl J Med. 2005; 352(20):2124-6. “Transplantation of umbilical-Cord Blood in babies with infantile Krabbe’s disease.”