Research and Clinical Trials

CAR T-Cell Immunotherapy for Central Nervous System Tumors (BrainChild)

Chimeric antigen receptor (CAR) T-cell immunotherapy is an experimental new cancer treatment that stimulates the immune system to fight disease.

Doctors and researchers at Seattle Children’s are testing CAR T-cell therapy in children and young adults with recurrent or refractory central nervous system (CNS) tumors (tumors of the brain and spine) including diffuse intrinsic pontine glioma (DIPG) and diffuse midline glioma (DMG).

These phase 1 trials are called BrainChild.

Learn how CAR T-cell immunotherapy works.

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

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

  • 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.

  • In this trial, the patient’s own T cells are reprogrammed to recognize and target the protein B7-H3, a surface molecule that is on most CNS tumors.

    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.

    This study is open to all types of CNS tumors that have recurred or progressed and for children with DIPG.

    This is the first CAR T-cell trial at Seattle Children's and one of the first in the United States for children with DIPG and is open to patients with DIPG any time after radiation.

    The study began in November 2019 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, -02 and -03 are phase 1 clinical 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 CAR T-cell therapy safe to give to children and young adults with relapsed or refractory CNS tumors (tumors of the brain or spinal cord)?
  • What is the best dose of intra-CNS–delivered CAR T cells for children and young adults with CNS tumors?
  • Does CAR T-cell therapy work against CNS tumors?

Who can join the BrainChild studies?

The BrainChild studies are for children and young adults who:

  •  Have recurrent or refractory CNS tumors:
    • BrainChild-01: tumor must express HER2
    • BrainChild-02: tumor must express EGFR
    • BrainChild-03 does not have a target expression requirement; it includes patients with DIPG
  • Are ages 1 through 26 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 these trials?

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

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 Seattle Children's Therapeutics at Seattle Children’s Research Division.

Meet Dr. Nicholas Vitanza (video: 0:45)

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 ALL: HA-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.
  • Solid tumors: The STRIvE trials are phase 1 trials testing CAR T-cell therapy in children and adolescents with relapsed or refractory solid tumors that express the protein EGFR or B7H3.
  • Osteosarcoma: ENLIGHTen-01
  • 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.

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Updated November 2021.