Research and Clinical Trials
T-Cell Immunotherapy for Brain and Central Nervous System Tumors
Seattle Children’s doctors and researchers are leaders in developing chimeric antigen receptor (CAR) T-cell immunotherapies to treat children and young adults with relapsed 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-01
An FDA-authorized clinical trial at Seattle Children’s is testing CAR T-cell therapy in children and young adults with relapsed or refractory brain and central nervous system (CNS) tumors who are not likely to survive with standard treatments. This trial is called BrainChild-01. DIPG (a type of brain tumor found at the base of the brain) is not included in this initial trial, but is planned to be included in future BrainChild trials.
In this trial, the patient’s own CAR 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 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 June 2018 and is currently enrolling patients.
What is the goal of the BrainChild-01 study?
BrainChild-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 BrainChild study, 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 BrainChild-01?
This study is for children and young adults who:
- Have relapsed or refractory brain or CNS tumors that express the protein HER2. It is not open to children with DIPG or other childhood cancers.
- Are ages 1 to 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 this trial?
- BrainChild-01: Dr. Nicholas Vitanza
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.
Are there studies for other childhood cancers?
- We are currently enrolling patients in phase 1 and 2 clinical trials testing CAR T-cell therapy in children and adolescents with relapsed or refractory acute leukemia or lymphoma (PLAT).
- We are currently enrolling patients in a phase 1 clinical trial testing CAR T-cell therapy in children and adolescents with recurrent or refractory neuroblastoma (ENCIT).
- Learn about our Brain Tumor Program.
- Read more about brain and spinal cord tumors.
- Read the complete BrainChild-01 protocol.
- Find more current research studies at Seattle Children’s or search ClinicalTrials.gov. Read our guide about searching for trials on ClinicalTrials.gov (PDF).
- Learn about the Ben Towne Center for Childhood Cancer Research and the Jensen Lab.
Updated May 2018.