Seattle Children’s doctors and researchers are leading efforts to better treat neuroblastoma in children and adolescents by boosting the immune system with immunotherapy.

Immunotherapy is a new cancer treatment that stimulates a child’s own immune system to fight disease. It is also known as targeted therapy or biotherapy.

Seattle Children’s is currently enrolling patients in a phase 1 clinical trial testing T-cell therapy in children and adolescents with recurrent or refractory neuroblastoma who are not likely to survive with current treatments. This trial is known as Engineered Neuroblastoma Cellular Immunotherapy (ENCIT-01). It is based on promising results from previous trials using immunotherapy for recurrent leukemia.

Doctors hope that when this new therapy is fully tested:

  • It will work quickly, so pediatric neuroblastoma treatment takes weeks, not years.
  • It will have milder side effects than other treatments, like chemotherapy.

What is T-cell therapy?

T cells are white blood cells in the immune system that fight infection. The goal with ENCIT is to reprogram a child’s own T cells so they can seek out and destroy cancer cells wherever they are hiding in the body.

The steps in this process are:

  1. A blood sample is drawn from the child at Seattle Children’s Hospital. This sample goes to a special part of Seattle Children’s Research Institute called the Therapeutic Cell Production Core. Lab staff remove the T cells from the sample, purify them and reprogram them. In this case, “reprogram” means to change the T cells by adding recombinant DNA (genetically modify them). Then, the newly programmed T cells are grown to multiply into millions of new cells.
  2. The changed T cells are put back into the child’s body through an intravenous (IV) infusion.
  3. The hope is that the changed cells will go to work right away, finding and destroying the cancer cells in the child’s body. The change tells the T cells to 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 cancer cells (a substance called L1CAM). When they bind, the T cells can attack the cancer cells as if they were fighting an infection.

The receptor that’s made on the T cells is called a chimeric antigen receptor (CAR). T cells that have the receptor may be called CAR T cells.

The change made to the T cells also “tags” the cells so our research team can track them in the body. If doctors want to stop the action of the T cells later, they can do this with the drug cetuximab, which recognizes the tag.

What are the goals of the study?

ENCIT-01 is a phase 1 trial. Phase 1 trials focus on finding out how much of a therapy to give, how to give it, how often to give it and when side effects occur.

Through the ENCIT study, researchers are working to answer these questions:

  • Is T-cell therapy safe to give to children and adolescents with recurrent or refractory neuroblastoma?
  • What is the largest dose that children and adolescents can stand (the maximum tolerated dose)?
  • Does T-cell therapy work against neuroblastoma?

Also, these clinical studies are meant to show that T cells taken from patients can be changed in the same way that T cells were changed in earlier laboratory studies.

Who can join the study?

The ENCIT study is for children and adolescents who:

  • Have high-risk neuroblastoma and have responded poorly to standard treatments (chemotherapy, radiation therapy or both)
  • Have had their neuroblastoma come back (a recurrence) after treatment
  • Are 1 to 18 years old

Researchers use many other factors to decide whether a patient can take part in a study (inclusion criteria) or cannot take part (exclusion criteria). The study team at Seattle Children’s can explain what these factors mean for you or your child.

Who is leading the ENCIT-01 phase 1 clinical trial?

Dr. Julie Park, a national leader in caring for children with neuroblastoma and in studying the disease, is leading the ENCIT-01 trial. She is a doctor in the Cancer and Blood Disorders Center at Seattle Children’s Hospital, and an investigator in the Center for Clinical and Translational Research at Seattle Children’s Research Institute.

Park led the most recent national Children’s Oncology Group (COG) trial for pediatric high-risk neuroblastoma and is the chair of the COG neuroblastoma committee, with a goal to advance treatment of high-risk neuroblastoma in children and adolescents.

ENCIT-01 uses a method developed by Dr. Michael Jensen of the Ben Towne Center for Childhood Cancer Research at Seattle Children’s Research Institute.

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Updated May 2017.

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