Research and Clinical Trials

CAR T-Cell Immunotherapy Clinical Trials for Brain and Spinal Cord Tumors

Seattle Children’s doctors and researchers are leaders in developing investigational chimeric antigen receptor (CAR) T-cell immunotherapies for children, teenagers and young adults with central nervous system (CNS) tumors, including DIPG.

CNS tumors are brain and spinal cord tumors, such as atypical teratoid/rhabdoid tumor (ATRT), diffuse intrinsic pontine glioma (DIPG) and diffuse midline glioma (DMG).

CAR T-cell immunotherapy is an experimental targeted cancer treatment that engineers changes in some cells of your immune system to fight disease.

Our phase 1 CAR T-cell clinical trials against CNS tumors are called BrainChild.

BrainChild-03 (targets B7-H3)

In the BrainChild-03 trial, a patient’s own T cells are reprogrammed to recognize and target the protein B7-H3, which is expressed on many pediatric CNS tumors but not healthy brain tissue.

The reprogrammed cells – now CAR T cells – will be put back into a patient’s brain through a catheter over multiple, repeated doses.

This study is open to patients 1-26 years of age with:

any type of malignant CNS tumors that has progressed or recurred
DIPG after initial radiation or after progression
DMG after initial radiation or after progression

The phase 1 portion of this trial is now completed, identifying the highest planned dose (100 million cells/dose) as tolerable. The FDA has approved an expansion cohort to allow more patients to enroll on this trial and spots remain for children with DIPG.

The study began in November 2019 and is currently recruiting patients.

Read more about the BrainChild-03 protocol at clinicaltrials.gov.
Learn more about this and other clinical trials at Seattle Children's.

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

BrainChild-04 (targets B7-H3, EGFR, HER2, and IL13Ra2)

In the BrainChild-04 trial, a patient’s own T cells are reprogrammed to recognize four targets (HER2, EGFR, B7-H3, and IL13Ra2) expressed on many pediatric CNS tumors but not healthy brain tissue. The reprogrammed cells – now CAR T cells – will be put back into a patient’s brain through a catheter over multiple, repeated doses.

This study is open to patients ages 1-26 years of age with:

any type of malignant CNS tumors that has progressed or recurred
DIPG after initial radiation or after progression
DMG after initial radiation or after progression

This is the first CAR T-cell clinical trial using CAR T cells that target four tumor targets.

The study began in April 2023 and is currently enrolling patients.

Read more about the BrainChild-04 protocol at clinicaltrials.gov.
Learn more about this and other clinical trials at Seattle Children's.

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

What is the goal of the CAR T-cell CNS clinical trials?

The BrainChild studies 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 intracranially-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 CAR T-cell CNS clinical trials?

The BrainChild studies are for children, teenagers and young adults with brain cancer (including ATRT, DIPG, DMG, ependymoma, ETMR, high grade glioma, medulloblastoma and pineoblastoma) who:

Have CNS tumors that have progressed or recurred
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 the CNS CAR T-cell clinical trials?

CNS CAR T-cell trial Lead: Dr. Nicholas Vitanza
BrainChild-03: Dr. Nicholas Vitanza
BrainChild-04: Dr. Rebecca Ronsley

T-cell trials at Seattle Children’s use methods developed by Seattle Children's Therapeutics at Seattle Children’s Research Division.

Are there CAR T-cell clinical trials 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, teens and young adults with relapsed or refractory acute leukemia or lymphoma.
Solid tumors:
- STRIvE-01 is a phase 1 trial testing CAR T-cell therapy in children, teens and young adults with relapsed or refractory solid tumors that express the protein EGFR or B7H3.
- ENLIGHTen-01 is a phase 1 clinical trial testing CAR T cells administered with UB-TT170, a small molecule fluorescein tag that targets folate receptors to mark tumors for clearance by CAR T cells, that is open to teens and young adults with recurrent or refractory osteosarcoma.
You can see many of Seattle Children's open clinical trials on our Research Studies page.

You can also 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).