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. Stem cell treatments may lower symptoms of the disease or condition that is being treated. 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. Stem cell implantation may help in strengthening the left-ventricle of the heart, as well as retaining the heart tissue to patients who have suffered from heart attacks in the past. Schwartz seeks to gain a better understanding of other brain diseases including autism. CHOC is the only hospital in the country with a focus on using immune-matched stem cells to treat enzyme deficiencies of the brain. Bone marrow stem cells, for example, differentiate primarily into blood cells. However, under natural circumstances somatic stem cells can become only a subset of related cell types. Without this enzyme, glycosaminoglycans build up and damage organs, including the brain. The UWMadison Stem Cell and Regenerative Medicine Center (SCRMC) operates under the School of Medicine and Public Health and the Office of the Vice. Stem cells from the blood and bone marrow are routinely used as a treatment for blood-related diseases. Individuals with this disease do not make an enzyme that helps break down long chains of sugar molecules called glycosaminoglycans. Children with this disease generally die before their 10th birthday. Schwartz’s starting point for treatment is Hurler’s Syndrome, a rare, inherited disease of metabolism called a lysosomal storage disorder. Stem cells are unspecified or partially specified cells which have the capacity to proliferate or self-renew and differentiate into a variety of cell types. 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.ĭr. Schwartz, PhD, a senior scientist at CHOC and research biologist at UC Irvine, has been involved in pediatric brain research for 25 years. Using Stem Cells to Treat Pediatric Brain Disorders
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