December 8, 2014
Stem cells contained in umbilical cord blood are used to treat a wide range of diseases and disorders, more than 80 to be specific. Some of the more commonly known diseases that can be treated are leukemia, severe anemia, and lymphoma, but the list goes on and on. One disease that isn’t as commonly known, or as regularly diagnosed, is Hurler Syndrome. This hereditary metabolic syndrome is a scary diagnosis for parents to hear. It is the most severe of the mucopolysaccharide storage diseases, which are caused by an inherited enzyme deficiency that is used to break down glycosaminoglycans, a vital bone- and tissue-building molecule. The symptoms of this disease are deformed bones and joints, mental impedance, short stature, breathing, hearing and heart problems, as well as an enlarged head. It causes a rapid deterioration of the child’s central nervous system and can be fatal, usually before the child’s 10th birthday, if not treated.
To treat this disease, a stem cell transplant must be done as early as possible, to halt the progression of the disease. Because it is a hereditary disease, an allogeneic transplant is necessary. Until recently, the child was dependent on finding a matching bone marrow donor, which, in some cases is difficult to impossible, depending on the patient. This led researchers to seek out and study a different source for the stem cells the patients needed and umbilical cord blood was the obvious choice.
Between the years of 1995 and 2002, researchers at Duke University performed cord blood transplants on 20 child patients (aged prenatal to 29 months) referred to them due to their Hurler’s Syndrome diagnosis1. The researchers tested each child and found matching cord blood donations. The donors of the cord blood units were unrelated to the patient and were not carriers of Hurler’s Syndrome. The patients received a conditioning treatment prior to transplant to prepare their bodies, as well as a prophylaxis to prevent graft vs. host disease. Following treatment and successful engraftment, the patients were evaluated by multiple specialists every 6-12 months to assess their mental and physical development and progression. The success of the trial was 85% after three years following engraftment. Of the 20 children 17 had survived (three passed away shortly after the treatment but from disease-related causes and not due to adverse reactions of the transplant.) In the time since the treatment, the growth rate of the surviving patients is normal and all the children have shown either stable or improved neurocognitive function. The researchers believe that cord blood is a superior treatment tool for Hurler’s Syndrome as compared to bone marrow stem cell transplants, which has shown a graft failure in 28 to 37 percent of like patients. The patients taking part in this clinical trial experienced improved health, growth and development, as well as prolonged survival.
One prior hurdle observed in treatment was the limited availability of compatible bone marrow stem cells. By using banked umbilical cord blood, which is becoming more and more available as more families choose to save this valuable resource, they saved time in searching and testing. Also, because cord blood stem cells do not have to be an exact genetic match to the patient, it made their use even more beneficial and an easier match.
This shows parents that whether it be a common or rare disease that affects your family, cord blood banking is a powerful and wise choice to help insure the future health of your family.
- Cord-Blood Transplants from Unrelated Donors in Patients with Hurler's Syndrome. Susan L. Staba, M.D., et al. The New England Journal of Medicine 2004; 350:1960-1969, DOI: 10.1056/NEJMoa032613. Published May 6, 2004. Found online http://goo.gl/XxJjJa.