CAR T-cell Therapy for Neuroblastoma
What You Need to Know Before You Apply
What is the purpose of this trial?
Patients with recurrent or refractory neuroblastoma are resistance to conventional chemotherapy. For this reason, the investigators are attempting to use T cells obtained directly from the patient, which can be genetically modified to express a chimeric antigen receptor (CAR). The CAR enables the T cell to recognize and kill the neuroblastoma cell through the recognition of CD171, a protein expressed of the surface of the neuroblastoma cell in patients with neuroblastoma. This is a phase 1 study designed to determine the maximum tolerated dose of the CAR+ T cells.
Will I have to stop taking my current medications?
The trial requires that you stop taking systemic corticosteroids (unless it's a small replacement dose) at least 7 days before joining. You also need to be off chemotherapy or biologic therapy for at least 7 days before starting the trial. If you're on anti-tumor antibody therapy, you need to wait 3 half-lives or 30 days, whichever is shorter, before enrolling.
Is CAR T-cell therapy safe for humans?
How is CAR T-cell therapy for neuroblastoma different from other treatments?
CAR T-cell therapy for neuroblastoma is unique because it uses genetically engineered T cells to specifically target and attack cancer cells, offering a personalized approach that harnesses the immune system. Unlike traditional treatments, this therapy can potentially provide long-term protection by persisting in the body and targeting tumor-associated antigens that are not widely present on normal tissues, reducing the risk of side effects.12346
What data supports the effectiveness of the treatment for neuroblastoma?
Research shows that CAR T-cell therapy, which uses specially modified immune cells, has been effective in shrinking tumors in children with neuroblastoma. Additionally, CAR T cells targeting low-density antigens have shown activity without causing harm, indicating potential safety and effectiveness.12467
Who Is on the Research Team?
Catherine Albert, MD
Principal Investigator
Seattle Children's Hospital
Are You a Good Fit for This Trial?
This trial is for children and young adults up to 26 years old with high-risk neuroblastoma or ganglioneuroblastoma that's resistant to standard treatments. They must have a life expectancy of at least 8 weeks, be in relatively stable health, and not have received certain recent therapies like allogeneic stem cell transplants.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Cell Generation
T cells are isolated, selected, transduced with a lentivirus to express CD171 CAR, and expanded in culture
Lymphodepletion and T Cell Infusion
Participants receive lymphodepletion chemotherapy followed by infusion of CAR+ T cells
Intensive Follow-up
Participants are monitored intensely for 6 weeks with serial blood testing and disease status re-evaluation
Long-term Follow-up
Participants are followed bi-annually for 5 years, then annually for 10 years to monitor long-term health effects
What Are the Treatments Tested in This Trial?
Interventions
- Patient Derived CD171 specific CAR T cells
Find a Clinic Near You
Who Is Running the Clinical Trial?
Seattle Children's Hospital
Lead Sponsor
Ben Towne Center for Childhood Cancer Research
Collaborator
The Evan Foundation
Collaborator