Seattle Children’s doctors and researchers are leaders in developing chimeric antigen receptor (CAR) T-cell immunotherapies to treat children and young adults with recurrent or refractory cancers.

Immunotherapy is an experimental new cancer treatment that uses the immune system to fight disease. Read more about how T-cell immunotherapy works.

CAR T-Cell Therapy for Brain and Central Nervous System Tumors: BrainChild

FDA-authorized clinical trials at Seattle Children’s are testing CAR T-cell therapy in children and young adults with recurrent or refractory brain and central nervous system (CNS) tumors who are not likely to survive with standard treatments. These trials are called BrainChild-01 and BrainChild-02. Diffuse intrinsic pontine glioma (DIPG), a type of brain tumor found at the base of the brain, is not included in these initial trials but is planned to be included in future BrainChild trials.

  • In this trial, the patient’s own T cells are reprogrammed to recognize and target the protein HER2, which is expressed by many pediatric brain tumors but not healthy brain tissue.

    The reprogrammed cells (now CAR T cells) will be put back into the patient’s body (infused) through a catheter, either into the place where the tumor has been removed or into the CNS ventricular system (intra-CNS). Placement will depend on the location of the tumor.

    BrainChild-01 is the first pediatric trial of intra-CNS CAR T-cell treatment in the United States.

    The study began in April 2018 and is currently enrolling patients.

  • In this trial, the patient’s own T cells are reprogrammed to recognize and target abnormal forms of the protein EGFR.

    This abnormal EGFR isn’t usually part of healthy brain tissue, but it is found on the surface of nearly half of pediatric brain cancers.

    The reprogrammed cells (now CAR T cells) will be put back into the patient’s body (infused) through a catheter, either into the place where the tumor has been removed or into the CNS ventricular system (intra-CNS). Placement will depend on the location of the tumor.

    The study began in November 2018 and is currently recruiting patients.

Contact Us

For more information, call 206-987-2106 or send us an email. Learn more about Seattle Children’s comprehensive Brain Tumor Program.

What is the goal of the BrainChild studies?

BrainChild-01 and BrainChild-02 are phase 1 trials. 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 BrainChild studies, researchers are working to answer these questions:

  • Is T-cell therapy safe to give to children and young adults with relapsed or refractory brain or CNS tumors? 
  • What is the best dose of intra-CNS–delivered CAR T cells for children and young adults with CNS tumors? 
  • Does T-cell therapy work against brain and CNS tumors?

Who can join the BrainChild studies?

The BrainChild studies are for children and young adults who:

  • Have recurrent or refractory brain or CNS tumors that express the protein HER2 or EGFR
  • Are ages 1 to 26 years old

At this time, the BrainChild studies are not open to children with DIPG or other non-CNS childhood cancers.

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 these trials?

Vitanza is a doctor in the Cancer and Blood Disorders Center at Seattle Children’s Hospital and an investigator in the Ben Towne Center for Childhood Cancer Research at Seattle Children’s Research Institute.

Meet Dr. Nicholas Vitanza (video: 2:17) 

Gust is an investigator in the Center for Integrative Brain Research at Seattle Children's Research Institute.

T-cell trials at Seattle Children’s use methods developed by Dr. Michael Jensen of the Ben Towne Center for Childhood Cancer Research and Dr. Julie Park of Seattle Children’s Research Institute.

Are there CAR T-cell studies for other childhood cancers?

  • Acute leukemia and lymphoma: The PLAT trials are phase 1 and 2 clinical trials testing CAR T-cell therapy in children and adolescents with relapsed or refractory acute leukemia or lymphoma.
  • AML, T-cell ALL and B-cell ALLHA-1 is a phase 1 clinical trial testing a type of T-cell therapy for children and young adults with relapsed or refractory acute leukemia or leukemia that does not respond to treatment after a donor stem cell transplant.
  • Neuroblastoma: ENCIT is a phase 1 clinical trial testing T-cell therapy in children and adolescents with recurrent or refractory neuroblastoma.
  • Solid tumors: STRIvE-01 is a phase 1 trial testing CAR T-cell therapy in children and adolescents with relapsed or refractory solid tumors that express the protein EGFR.
  • See many of Seattle Children's open clinical trials on our Current Research Studies page.
  • Or search by diagnosis for many clinical trials available through Seattle Children's and our partners on ClinicalTrials.gov. Read our guide about searching for trials on ClinicalTrials.gov (PDF).

Contact Us

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

Learn More

Updated November 2018.

New Cure. New Hope.

Strong Against Cancer logoThrough our Strong Against Cancer initiative, we’re leading the fight against childhood cancer with immunotherapy. Learn how you can get involved.