CAR T Cell Therapy for Pediatric Brain Cancer
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment for children and young adults with specific brain tumors that recur or resist standard treatments. It uses HER2-specific CAR T cell locoregional immunotherapy, which modifies a person’s immune cells to better attack cancer cells in the brain. Researchers aim to determine if this treatment, delivered directly into the brain, can safely and effectively target HER2-positive tumors (a specific protein found in some cancer cells). Children or young adults whose brain tumors have returned and tested positive for HER2, and who already have a special catheter in place for treatment, might be suitable candidates for this trial. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the chance to be among the first to receive this new therapy.
Will I have to stop taking my current medications?
The trial protocol does not specify if you must stop taking all current medications, but you must stop certain treatments like chemotherapy, biologic therapy, and anti-tumor antibody therapy for specific periods before enrolling. Corticosteroid treatment must be stable or decreasing, with a maximum dose specified.
Is there any evidence suggesting that this treatment is likely to be safe for humans?
Research has shown that HER2-specific CAR T cell therapy could be promising for treating solid tumors, including brain tumors. In these studies, patients generally tolerated the therapy well. For instance, one study found that patients with glioblastoma and other cancers experienced manageable side effects, indicating the treatment is likely safe.
Moreover, similar CAR T cell treatments targeting other proteins have been shown to be safe and potentially effective for some patients. These studies involved administering the treatment directly to the brain, which is similar to the approach in this trial.
As a Phase 1 study, the main focus is on safety and the body's response to the treatment. So far, data suggests that HER2-specific CAR T cell therapy can be administered safely, but careful monitoring remains important with any new treatment.12345Why do researchers think this study treatment might be promising?
Unlike the standard treatments for pediatric brain cancer, which often involve surgery, radiation, and chemotherapy, HER2-specific CAR T Cell Locoregional Immunotherapy introduces a novel approach by harnessing the body's own immune system to target cancer cells. This therapy uses genetically engineered T cells that are designed to specifically attack HER2-positive cancer cells, which is a new mechanism of action compared to traditional methods. The treatment is delivered directly into the tumor site or ventricular system, allowing for a targeted approach that minimizes damage to surrounding healthy tissue. Researchers are excited about this therapy because it offers the potential for a more precise and less invasive treatment, with the hope of improved outcomes and fewer side effects for young patients.
What evidence suggests that HER2-specific CAR T Cell Locoregional Immunotherapy might be an effective treatment for pediatric brain cancer?
Research shows that a new treatment called HER2-specific CAR T cell therapy holds promise for treating brain tumors in children. This trial will evaluate two delivery methods for this therapy. In ARM A, participants with supratentorial tumors will receive CAR T cells directly into the tumor resection cavity. In ARM B, participants with infratentorial or leptomeningeal tumors will receive CAR T cells into the fourth ventricle or lateral ventricle, respectively. This therapy uses specially modified immune cells to target and attack tumor cells with a marker called HER2, often found in certain brain cancers. Early studies suggest that delivering these modified cells directly to the brain is safe and doesn't cause serious side effects. Evidence also indicates that this treatment can help manage progressive glioblastoma, a type of brain cancer. While more research is needed, these early results offer hope for treating challenging brain tumors in children and young adults.23678
Who Is on the Research Team?
Rebecca Ronsley, MD
Principal Investigator
Seattle Children's Hospital
Are You a Good Fit for This Trial?
This trial is for children and young adults aged 1 to 26 with recurrent or refractory HER2-positive CNS tumors who have no standard therapy options left. They must have a CNS catheter in place, a life expectancy of at least 8 weeks, recovered from previous treatments, stable or decreasing steroid use, good organ function and lab values, not be pregnant or breastfeeding, and agree to use contraception.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment Course 1
Participants receive weekly doses of CAR T cells for three weeks, followed by a week off and an examination period
Treatment Course 2
Participants receive another course of weekly doses of CAR T cells for three weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment, including MRI evaluations
Extension
Participants may continue receiving additional courses of CAR T cells if no adverse effects are observed and T cells are available
What Are the Treatments Tested in This Trial?
Interventions
- HER2-specific CAR T Cell Locoregional Immunotherapy
Find a Clinic Near You
Who Is Running the Clinical Trial?
Seattle Children's Hospital
Lead Sponsor