Immunotherapy Program

A World Leader in Cellular Immunotherapy

Seattle Children’s is an international leader in treatment and research focused on curing childhood disease using the immune system (immunotherapy). Experts in our Immunotherapy Program work to improve outcomes for young people and reduce side effects of treatment by harnessing the power of the immune system.

Our Cancer and Blood Disorders Center offers state-of-the-art therapies and investigational clinical trials often not available at other hospitals. Many of these promising immunotherapies are chimeric antigen receptor (CAR) T-cell therapies for cancer. We also design and develop other immune effector cell therapies for life-threatening and debilitating childhood diseases.

Cellular Immunotherapy Clinical Trials for Cancer and Other Childhood Conditions

By leading and taking part in clinical trials for promising new immunotherapies, we can offer innovative therapies to children with life-threatening or life-limiting illnesses. Developing novel immunotherapies at Seattle Children’s allows us to rapidly translate our own research and development to patient care after Food and Drug Administration (FDA) approval. Seattle Children’s Therapeutics, part of Seattle Children’s Research Division, is devoted to envisioning and testing next-generation cell and gene therapies for cancer and other pediatric conditions.

Seattle Children’s was one of the first in the world to offer CAR T-cell trials for children with various types of leukemia, solid tumors, diffuse intrinsic pontine glioma (DIPG) brain tumors and now children with systemic lupus erythematosus (SLE).

More than 600 children, teens and young adults have received investigational CAR T-cell therapy through clinical trials at Seattle Children’s and at our collaborating sites. CAR T-cell products developed at Seattle Children’s Therapeutics are made onsite at our own state-of-the-art Therapeutics Cell Manufacturing facility. This means we can make and deliver treatments quickly to children we treat.

Learn about our clinical trials of cell and gene therapies for children, teens and young adults

• Leukemia and lymphoma
• Brain and spinal cord tumors
• Solid tumors
• Autoimmune disorders:
  • The REACT-01 (Reversing Autoimmunity Through Cell Therapy) trial is a phase 1 study for children and young adults ages 2 to 30 with treatment-resistant systemic lupus erythematosus (SLE). In this study, the person’s T cells are reprogrammed to recognize and target a B-cell marker called CD19. Read more about the first CAR T-cell clinical trial for children with lupus.
  • The RESET-Myositis trial (CAB-201-002) is a phase 1/2 trial for children ages 6 to 17 with treatment-resistant juvenile idiopathic inflammatory myositis (JIIM). In this study, the person’s T cells are reprogrammed to recognize and target a B-cell marker called CD19. Read about RESET-Myositis at ClinicalTrials.gov.

Learn more about the clinical trials Seattle Children’s offers. Search by diagnosis for many clinical trials available through Seattle Children’s and our partners on ClinicalTrials.gov.

FDA-Approved CAR T-Cell Therapy

Seattle Children’s offers CAR T-cell therapy for eligible patients up to the age of 25 with B-cell acute lymphoblastic leukemia that has not responded to or has returned after initial treatment. This CAR T-cell therapy, known as KYMRIAH™, was approved by the FDA in 2017. It works by genetically altering the body’s T-cells — immune system cells — to detect and destroy infected or cancerous cells.

How does the CAR T-cell therapy process work?

CAR T-cell therapy is the best-known type of cellular immunotherapy and is a promising treatment for some childhood cancers and other disorders. It works by teaching T cells, a type of white blood cell, to seek out and destroy cancer cells wherever they are hiding in the body. Here are the 5 steps in the CAR T-cell process:

1. If the CAR T cells are made from your own T cells, we do a procedure called apheresis. In this procedure, a machine is used to isolate T cells from your child’s blood. This takes a few hours. Some CAR T cells are made from donor T cells rather than your own T cells.
2. The T cells are purified and re-educated. In this case, “re-educate” means to change the T cells by adding recombinant DNA (genetically modify them) to recognize and destroy the target disease cell. Then, the newly re-educated T cells multiply into millions of new cells.
3. For most diseases, the changed T cells, now CAR T cells, are put back into your child’s body through an intravenous (IV) infusion. To treat brain tumors, the cells are injected in the area of the disease.
4. The hope is that the CAR T cells will go to work right away, finding and destroying the disease or cancer cells in your child’s body. The CAR T cells make a place on their surface (a receptor) that acts like Velcro. This receptor allows the T cells to recognize and bind to a target on the disease or cancer cells. When they bind, the CAR T cells can attack the target cells as if they were fighting an infection.
5. Our care team watches your child closely and is specially trained to identify and manage any side effects. Complications are generally temporary and resolve with treatment. Possible side effects may be mild or severe. These include cytokine release syndrome, an inflammatory condition with flu-like symptoms like high fever or chills, and neurological problems such as confusion or difficulty with language.