Money will be used to develop a treatment for mucopolysaccharidosis.

A project from CHOC Children’s Research Institute has been awarded a $5.5 million grant from the California Institute for Regenerative Medicine (CIRM). The grant will be used to develop a stem cell-based therapy for mucopolysaccharidosis (MPS I), a metabolic disease that causes neurodegeneration and defects in other major organ systems.

Children with inherited degenerative diseases of the brain are expected to be among those to benefit from novel approaches based on stem cell therapy, which has reportedly been shown to work in the milder forms of similar diseases that do not affect the brain.

“While uncommon, pediatric genetic neurodegenerative diseases account for a large burden of mortality and morbidity in young children,” notes Philip H. Schwartz, Ph.D., senior scientist at the CHOC Children’s Research Institute and managing director of the facility’s National Human Neural Stem Cell Resource, who will oversee the project. “Hematopoietic stem cell transplant (HSCT) can improve some non-neural symptoms of these diseases but does not treat the deadly neurodegenerative process.

“Our approach—targeting the effects of the disease on organs besides the brain with HSCT and neurodegeneration with a second stem cell therapy specifically designed to treat the brain—is a strategy for whole-body treatment of MPS I,” Dr. Schwartz explains. “Our approach is also designed to avoid the need for immunosuppressive drugs to prevent rejection of the transplanted cells.” This research is designed to address two issues: early intervention is required and possible in this patient population; and teaching the immune system not to reject the transplanted cells is required.

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