CHOC Children's research project receives $5.5M CIRM grant

May 30 2012

A CHOC Children's research project, under the direction of 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, 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 the treatment of mucopolysaccharidosis (MPS I), a fatal metabolic disease that causes neurodegeneration, as well as defects in other major organ systems.

Based on a number of medical and experimental observations, children with inherited degenerative diseases of the brain are expected to be among the first to benefit from novel approaches based on stem cell therapy (SCT).

1. These diseases currently lack effective therapies and can cause profound mental retardation or lead to death.
2. SCT has already been shown to work in the milder forms of similar diseases that do not affect the brain.
3. Experimental work and early clinical studies have clearly shown that stem cells delivered directly into the brain can be used to treat diseases affecting the brain.
4. The clinical safety of stem cells delivered directly into the brain has already been established during recent Phase 1 clinical trials.

Dr. Schwartz explains, "While uncommon, pediatric genetic neurodegenerative diseases account for a large burden of mortality and morbidity in young children. Hematopoietic (bone marrow) 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. Our approach is also designed to avoid the need for immunosuppressive drugs to prevent rejection of the transplanted cells."

This research is designed to lead to experimental therapy, based on stem cells, by addressing two critical issues: early intervention is required and possible in this patient population; and "teaching" the immune system not to reject the transplanted cells is required. This research also sets the stage for efficient translation of this technology into clinical practice, by adapting transplant techniques that are standard in clinical practice or in clinical trials, and using laboratory cell biology methods that are easily transferrable to clinical cell manufacturing.

Nationally recognized for his work in the stem cell field, Dr. Schwartz' research focuses on the use of stem cells to understand the neurobiological causes of autism and other neurodevelopmental disorders.