CAR T-cell Therapy for Neuroblastoma
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called CAR T-cell therapy for individuals with neuroblastoma, a cancer that often resists standard treatments. The goal is to assess the safety and effectiveness of using the patient's own immune cells, modified to target the cancer. Participants will receive these modified cells after undergoing chemotherapy to prepare their bodies. The trial seeks individuals with neuroblastoma that has returned or persisted despite other treatments. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative therapy.
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 there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that CAR T-cell therapy, which targets CD171 for treating neuroblastoma, appears safe. Studies have found that targeting CD171 does not harm important organs in animal tests with rhesus macaques. This suggests the treatment might not lead to serious side effects in vital organs.
In early human tests, CAR T-cell therapy for neuroblastoma has been safe and feasible, though scientists continue to work on enhancing its effectiveness. It is important to note that this treatment remains in the early stages of human testing, so researchers are determining the optimal dose.
Overall, while early studies have shown the therapy to be well-tolerated, more research is needed to fully understand its safety in humans as it progresses through clinical trials.12345Why are researchers excited about this trial's treatments?
Researchers are excited about CAR T-cell therapy for neuroblastoma because it represents a personalized approach that directly targets cancer cells. Unlike traditional treatments like chemotherapy or radiation, which can affect both healthy and cancerous cells, CAR T-cell therapy uses a patient's own immune cells, engineered to recognize and attack neuroblastoma cells specifically. This treatment involves modifying T cells to express CD171-specific CARs, which allow them to identify and destroy cancer cells more efficiently. Additionally, the therapy includes variations in generation and spacer length, which researchers believe may enhance the effectiveness and persistence of the T cells in the body. These innovative features give hope for improved outcomes with potentially fewer side effects compared to conventional therapies.
What evidence suggests that this trial's treatments could be effective for neuroblastoma?
Research has shown that a new treatment using CD171-specific CAR T cells could help treat neuroblastoma, a type of cancer. CD171 is a protein found on many neuroblastoma cells, making it a good target for treatment. Early lab studies demonstrated that these CAR T cells can successfully attack neuroblastoma cells by recognizing this protein. They have also been effective against similar proteins in other types of solid tumors, suggesting potential benefits for neuroblastoma patients. This trial will evaluate different generations of CD171-specific CAR T cells, with participants receiving either 2nd or 3rd generation CAR T cells. While these results are promising, further research is needed to confirm their effectiveness in people with neuroblastoma.23678
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