Stem Cell Research for Kids

The Importance of Stem Cell Research

Stem cells have unique properties. First, researchers can grow lots of them in the lab, making them easily available. Second, because they are immature, they can mature into many different cell types in the body opening up a world of possibilities for the treatment of diseases.

What are stem cells?

Stem cells are a unique cell type that can develop into many different types of cells including red blood cells that carry oxygen, white blood cells that fight infections and platelets that help blood clot. Because they have the potential to develop into many different cell types in the body, stem cells can serve as a repair system for the body.

Where do stem cells come from?

Doctors can get the stem cells from:

• bone marrow (this is also called a bone marrow transplant)
• bloodstream
• umbilical cord blood after the cord is no longer attached to a newborn baby

Stem Cell Treatments and Transplants

Stem cell treatments and transplants can help people with severe blood or immune system illnesses, some kinds of cancer, immune deficiencies and autoimmune diseases such as lupus, and blood disorders (such as thalassemia or sickle cell disease).

At CHOC, we participate in both international and national clinical trials and offer innovative targeted therapies, immunotherapy as well as a robust stem cell transplant program to treat different conditions in children.

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.