SCI Faculty Troy Lund, MD, PhD refines decision-making process for hematopoietic cell transplant treatment of Hurler's Syndrome
Hurler's Syndrome is caused by a mutation in a gene (IDUA) that encodes an enzyme that is responsible for clearing cellular waste products, and hence prevents accumulation of toxic byproducts of cellular function. Patients lacking IDUA experience a wide array of developmental and neurological dysfunction that affects their intellect, organ development and growth. These clinical manifestations can be reduced by replacement of the missing enzyme through hematop[oietic cell transplants (HCT) from donors who have the normal gene and functional enzyme.
Stem Cell Institute faculty member Troy Lund, MD,PhD and his colleagues in the Pediatrics department asked whether or not the source of hematopoietic cells used for transplants makes a difference regarding the patients' ability to clear the toxic substances into their urine up to 28 years after their transplant. They found that the two sources of transplanted cells had similar effects, and concluded that other factors, like HLA matching, cell dose, availability of an appropriate donor, and the timing of the transplant are more important considerations when making decisions about HCT cell source to treat Hurler's syndrome. This is good news for Hurler's syndrome patients since it increases the chances that an appropriate donor can be found during the crucial time for transplant around their first birthday.