Cellular Immunotherapy Clinical Trials for Brain and Spinal Cord Tumors
Seattle Children’s doctors and researchers are leaders in developing new therapies that stimulate the immune system (immunotherapies) to treat children, teens and young adults with central nervous system (CNS) tumors. CNS tumors are brain and tumors, such as atypical teratoid/rhabdoid tumor (ATRT), diffuse intrinsic pontine glioma (DIPG) and diffuse midline glioma (DMG).
Many of these promising immunotherapies are T-cell therapy, some developed by Seattle Children’s Therapeutics. Read about how T-cell immunotherapy works. We also develop and study other such as and .
What immunotherapy clinical trials are open for patients with brain and spinal cord tumors?
These clinical trials authorized by the Food and Drug Administration (FDA) study new treatments for children and adults with or brain and spinal cord tumors. For more information, call 206-987-2106 or send us an email at [email protected].
The study team at Seattle Children’s can explain the factors that determine if a patient can take part in a study (inclusion criteria) or cannot take part (exclusion criteria).
- The BrainChild-03 trial is a phase 1 trial for children and young adults ages 1 to 26 with either nonpontine DMG or DIPG. In this study a patient’s T cells are reprogrammed to recognize and target the protein B7-H3. This protein is expressed on many pediatric CNS tumors but not healthy brain tissue. The reprogrammed CAR T cells will be put back into a patient’s brain through a catheter over multiple repeated doses. Read more about the BrainChild-03 protocol at ClinicalTrials.gov. Learn about CAR T-Cell Immunotherapy for Brain and Spine Tumors (PDF).
- The PNOC028 trial is a phase 1 study for children and young adults ages 1 to 39 with recurrent or progressive malignant brain tumors. In this study, reprogrammed from a healthy donor are injected into the tumor or the area where the tumor was removed, using a device (Ommaya reservoir) placed under the scalp. Read about PNOC028 on ClinicalTrials.gov.
What are the goals of the CNS 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 what side effects occur. Phase 2 trials look at whether a therapy is safe and whether it works in people who have a certain disease.
Doctors hope that when these new therapies are fully tested, they will:
- Work quickly, so treatment takes weeks, not years
- Have milder side effects than other treatments, like chemotherapy
Are there immunotherapy clinical trials for other childhood diseases?
We offer many trials focused on using the immune system to better treat children, teens and young adults with diseases such as:
- Leukemia and lymphoma.
- Solid tumors.
- Systemic lupus erythematosus (REACT-01). Read about the first U.S. CAR T-cell clinical trial for children with lupus.
See many of Seattle Children’s open clinical trials:
- On our Current Research Studies
- Search by diagnosis for many clinical trials available through Seattle Children’s and our partners on ClinicalTrials.gov.
Contact Us
For more information, call 206-987-2106 or send us an email at [email protected].
Learn More
- Learn about our Brain Tumor Program.
- Learn about our Diffuse Intrinsic Pontine Glioma Research Program.
- Read more about brain and spinal cord tumors.
- See an overview of our Immunotherapy Program.
- Learn about Seattle Children’s Therapeutics.
Updated June 2026
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