University of South Florida researchers led the preclinical study exploring a human cell therapy for Sanfilippo syndrome
Tampa, FL (April 1, 2014) — A University of South Florida-led study provides new insight into treating an inherited metabolic disorder that causes serious neurological and behavioral disabilities in children, usually leading to death in the teen years.
The preclinical study explored the effects of human umbilical cord mononuclear cells (hUCB MNCS) injected to counter the symptoms and progression of Sanfilippo syndrome type III B (MPS III B). Researchers found that repeated injections into laboratory mice modeled with the disorder showed clear benefits for the mice receiving multiple injections, compared to mice receiving either a single high or low dose.
Alison Willing, PhD
The study will be published in a future issue of Cell Transplantation but is currently freely available on-line as an unedited early e-pub at: http://www.ingentaconnect.com/content/cog/ct/pre-prints/content-ct1121willing.
MPS III B results from a genetic deficit of the Naglu enzyme. This missing enzyme creates a build-up of the complex carbohydrate heparan sulfate, which accumulates in lysosomes, cells responsible for waste disposal. With MPS III B, accumulations of heparan sulfate inside cells cause damage to multiple organs, including the brain.
“Cell therapy has recently received attention as a potential treatment for lysosomal storage diseases,” said study lead author Alison E. Willing, PhD, a professor in the Department of Neurosurgery and Brain Repair, USF Health Morsani College of Medicine. “We have previously shown that a single hUBC injection into the cerebral ventricle of pre-symptomatic MPS III B mice, or intravenous cell delivery at different disease stages, was beneficial to the enzyme-deficient mice. In the current study, we examined whether administering repeated doses of hUBC MNCs would have a greater effect than a single dose and help to prevent progressive neurodegeneration.”
