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.

Learn how CAR T-cell therapy works.

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.

Read the complete ENCIT-01 study protocol at clinicaltrials.gov.

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.

Contact Us

For more information, please call 206-987-2106 or send us an email.

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Updated April 2018.

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