The procedure that Lauren needs is a surgery during which stem cells from the umbilical cord blood of her healthy brother, Nathan, will be used to correct her defect.
The hope is that these stem cells, once introduced into Lauren’s bone marrow, will differentiate into her existing systems and begin producing the enzyme she lacks, called heparan sulfate. The enzyme is critical to the breakdown of mucopolysacchrides (complex sugar molecules) that would otherwise collect in her tissues, causing progressive and permanent damage.
Stem cells have proven to be the most effective way of treating Sanfilippo Syndrome, but the procedure is still experimental. Stem cells are used in favor of adult bone marrow, because these young cells are more easily adaptable to their new environment and more likely to differentiate into the patient’s systems and produce the needed enzyme.
An article from the Duke University Medical Center, where the procedure is being done states “Indeed, it appears that donor stem cells inexplicably home in on defective tissue and establish themselves there, as though they are missionaries recruited to rescue cells in need, the scientists said.” and “Just a few donor stem cells are enough to provide a wide swath of damaged tissue with the enzyme necessary to restore function, the researchers believe.”
The final success of the procedure comes when the stem cells “traverse their way from the bloodstream into the brain, where they eventually engraft and differentiate into the needed types of brain cells.” Which halts the damage.
The introduction of stem cells into a patient can be repeated over time if not immediately successful.
