Reports of Stem Cell Transplant for treatment in Hurler Syndrome and Leukemia

Two patient cases reported recently needing stem cell transplants to treat their condition.

The first story is about a 1 year old boy Justin Wolford with Hurler syndrome who gets an 8 hour weekly enzyme replacement injections at Hershey Medical Centre. However, the enzyme injection is only temporary and the only way to slow the progression of the disease (85% chance of stopping) is a stem cell transplant. Hurler syndrome (otherwise known as muccopolysaccharidosis Type 1) is debilitating on the growth of children and are progressively neurologically impaired with a possibility of early death. Gone undetected, he could have been faced with a life of progressing mental retardation, hearing loss, clouding of the eyes and spinal deformities. Eventually, the mother said, his organs would have begun to shut down.

Unfortunately, his only 7 year old sister was not a match and a matching unit is in the process of being found.

Malaysia has a number of Hurler syndrome sufferers (US estimates of incidence is 1 in 25,000 but I couldn't find any numbers for Malaysia) and I wonder if they are aware that with an early diagnosis and stem cell transplant, their child might have a decent chance of living quite normally. StemLife did have one enquiry for this condition, but the child was already of significant age and there was a significant risk associated with the procedure.

The next story is about an 8 year old boy, Zachary McCracken, who has been fighting acute lymphoblastic leukemia for three years but relapsed and received an umbilical cord blood stem cell transplant last week. Three months ago, the McCrackens were hoping to find a bone marrow match for the boy. Hundreds of the boy's neighbours donated blood during a December fund drive at the boy's school, but the family was unable to find the right match. They had little choice but to search for a suitable unit of cord blood, but the article does not specifiy where they obtained the unit. The transplant was conducted at Duke University Hospital and the parents will wait out the 4-6 months until their son is able to leave the isolation room.

Zachary McCracken and his recovery is available online. Fingers crossed for both these young patients. The StemLife team hopes they are able to return home soon.