Using Stem Cells to Treat Pediatric Brain Disorders
Philip H. Schwartz, PhD, a senior scientist at CHOC and research biologist at UC Irvine, has been involved in pediatric brain research for 25 years. Dr. Schwartz and his team use stem cell research not only to treat disease, but also to gain a better understanding of disease processes– working to unlock mysteries that will eventually become treatments and one day, cures.
Dr. Schwartz’s starting point for treatment is Hurler’s Syndrome, a rare, inherited disease of metabolism called a lysosomal storage disorder. Children with this disease generally die before their 10th birthday.
Individuals with this disease do not make an enzyme that helps break down long chains of sugar molecules called glycosaminoglycans. Without this enzyme, glycosaminoglycans build up and damage organs, including the brain. CHOC is the only hospital in the country with a focus on using immune-matched stem cells to treat enzyme deficiencies of the brain.
Through research, Dr. Schwartz seeks to gain a better understanding of other brain diseases including autism. Dr. Schwartz uses Induced Pluripotent Stem Cell (iPSC) technology to transform skin stem cells to brain stem cells, allowing him to study brain disease without ever touching the brain. Dr. Schwartz and his team built a cell production facility at CHOC and his next step is to convert it into a FDA-compliant cell manufacturing operation, where one day the stem cells can be transplanted into children, providing them, for the first time, a comprehensive treatment that has the possibility of curing them.
