CAR T-Cell Therapy for Pediatric Brain Cancer
Trial Summary
What is the purpose of this trial?
This phase I trial investigates the side effects of chemotherapy and cellular immunotherapy in treating children with IL13Ralpha2 positive brain tumors that have come back after a period of improvement (recurrent) or do not respond to treatment (refractory). Cellular immunotherapy (IL13(EQ)BBzeta/CD19t+ T cells) are brain-tumor specific cells that may induce changes in body's immune system and may interfere with the ability of tumor cells to grow and spread. Chemotherapy drugs, such as as cyclophosphamide and fludarabine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Many patients with brain tumor respond to treatment, but then the tumor starts to grow again. Giving chemotherapy in combination with cellular immunotherapy may kill more tumor cells and improve the outcome of treatment.
Do I have to stop taking my current medications for the trial?
The trial protocol does not specify if you must stop taking your current medications. However, there are specific 'washout' periods (time without taking certain medications) required for some treatments before starting the study. It's best to discuss your current medications with the study team to understand any necessary adjustments.
What data supports the effectiveness of this treatment for pediatric brain cancer?
Research shows that IL13Rα2-targeted CAR T cells have demonstrated promising anti-tumor activity in glioblastoma (a type of brain cancer) models, with improved survival in mice and some clinical benefits in pediatric patients. Additionally, CD19 CAR T cells have shown high remission rates in leukemia, suggesting potential for similar therapies in other cancers.12345
Is CAR T-cell therapy safe for pediatric brain cancer?
CAR T-cell therapy has shown potential in treating certain cancers, but it can cause side effects like cytokine release syndrome (CRS) and neurotoxicity, which can be serious but are often manageable with medical care. Safety data from other conditions suggest that while there are risks, they can be mitigated with careful monitoring and treatment.13678
How is CAR T-Cell Therapy for Pediatric Brain Cancer different from other treatments?
This treatment is unique because it uses specially engineered T-cells to target a specific protein (IL13Rα2) found on brain cancer cells, which is not present in normal brain tissue, potentially reducing side effects. Additionally, the therapy is delivered directly into the brain's ventricles, which may enhance its effectiveness compared to traditional systemic treatments.12457
Research Team
Leo D Wang
Principal Investigator
City of Hope Medical Center
Eligibility Criteria
This trial is for children with IL13Ralpha2 positive brain tumors that have returned or are not responding to treatment. They must have good kidney function, no severe infections, and a certain level of physical ability despite their illness. Children who are pregnant, breastfeeding, or unable to follow the study procedures cannot participate.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Lymphodepletion
Patients receive cyclophosphamide and fludarabine intravenously to prepare for CAR T cell therapy
CAR T Cell Treatment
Patients receive autologous IL13(EQ)BBzeta/CD19t+ T cells intraventricularly once a week for up to 4 cycles
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Cyclophosphamide
- Fludarabine
- IL13Ralpha2-specific Hinge-optimized 41BB-co-stimulatory CAR Truncated CD19-expressing Autologous T-Lymphocytes
Cyclophosphamide is already approved in United States, European Union, Canada, Japan for the following indications:
- Breast cancer
- Ovarian cancer
- Multiple myeloma
- Leukemia
- Lymphoma
- Rheumatoid arthritis
- Breast cancer
- Ovarian cancer
- Multiple myeloma
- Leukemia
- Lymphoma
- Rheumatoid arthritis
- Breast cancer
- Ovarian cancer
- Multiple myeloma
- Leukemia
- Lymphoma
- Rheumatoid arthritis
- Breast cancer
- Ovarian cancer
- Multiple myeloma
- Leukemia
- Lymphoma
Find a Clinic Near You
Who Is Running the Clinical Trial?
City of Hope Medical Center
Lead Sponsor
National Cancer Institute (NCI)
Collaborator