Using Cord Blood to Treat Babies with Hurler Syndrome
Do you have a family history of Hurler Syndrome? Are you currently pregnant or planning to start a family? There is positive news for treatment of this rare, inherited and life-threatening disease.
About Hurler Syndrome
Hurler syndrome is also known as mucopolysaccharidosis type I (MPS I). According to the National MPS Society, this disease is “relentlessly progressive and potentially fatal.” It is a rare, inherited disease of metabolism in which a person is missing an enzyme needed to break down long chains of sugar molecules called glycosaminoglycans (formerly called mucopolysaccharides). These molecules help the body build bones and tissue. Since the body cannot break down these large molecules, a buildup occurs which damages organs and tissues. Untreated, this often results in permanent brain damage and childhood death.
Hope with Cord Blood Treatment
There is some good news. The use of umbilical cord blood in Hurler Syndrome treatment for affected babies under nine months old is showing positive outcomes in initial trials. The study was conducted by the Children’s Hospital of Pittsburgh at the University of Pittsburgh Medical Center, with funding by the Caterina Marcus Foundation. According to the study, allogeneic hematopoietic stem cells from the transplant of cord blood provides a source for the normal enzyme that is donated to the deficient cells. This decreases the accumulation of glycosaminoglycan sugar molecules.
Cord Blood Banking for Inherited Diseases
The key for treating children with Hurler Syndrome is early detection. This can be problematic since symptoms may not appear before the age of nine months, when the treatment would be most effective. Also, standard screenings aren’t widely used for Hurler Syndrome.
Since Hurler Syndrome is an inherited disease, knowing your family history further is key. It also highlights the need to start banking cord blood with your first child. Like other disorders that rely on allogeneic treatments, the cord blood stem cells must come from a healthy sibling or matched unrelated donor vs. from oneself. Like all transplants with this disease, it is not a guaranteed cure, and can have mixed outcomes. Still, if babies or very young children in your family have been affected by Hurler Syndrome or any of the six other MPS diseases, it makes good sense to speak to your healthcare provider and get more information as soon as possible.