GD2-targeted CAR T-Cells for Neuroblastoma
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment that uses special immune cells, called T cells, to fight cancer. Researchers modify these T cells with a gene to better recognize and attack cancer cells with a marker called GD2. This treatment is known as C7R-GD2.CART cells. The study aims to determine the safest dose of these modified T cells and their effectiveness in shrinking tumors. Suitable candidates for this trial include individuals with neuroblastoma or certain other cancers, such as breast cancer or melanoma, whose cancer has either returned or not responded to previous 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 new therapy.
Will I have to stop taking my current medications?
The trial does not specify if you must stop all current medications, but you cannot be on immunosuppressive drugs or investigational drugs. Maintenance therapy with certain non-investigational oral cancer drugs is allowed up to 48 hours before the infusion.
Is there any evidence suggesting that this trial's treatment is likely to be safe?
Research has shown that GD2-targeted CAR T cells could effectively treat certain cancers. One study administered these cells to patients and found them generally safe. No serious side effects occurred, and only 15.4% of patients experienced mild side effects, such as a low-grade fever. Another study discovered that these cells remained in the body for over a year in some patients, suggesting long-term cancer-fighting potential.
However, adding a new gene called C7R to the GD2-CAR T cells to extend their lifespan led to different side effects. In one patient group, 88% experienced mild nerve-related symptoms, which could be managed with treatment.
Overall, these studies suggest that while some side effects exist, the treatment is generally well-tolerated. Ongoing research aims to determine the safest dose and understand how long these cells remain active in the body.12345Why do researchers think this study treatment might be promising?
Most treatments for neuroblastoma, like chemotherapy and radiation, target rapidly dividing cells but can also harm healthy tissues. In contrast, C7R-GD2.CART cells are unique because they are engineered T-cells that specifically target GD2, a molecule found on neuroblastoma cells, providing a more precise attack on cancer. This targeted approach is promising because it can potentially lead to fewer side effects compared to traditional therapies. Additionally, the treatment is administered without lymphodepletion chemotherapy, which might reduce the overall treatment burden for patients. Researchers are excited about these CAR T-cells because they represent a novel way to harness the immune system's power to fight cancer more effectively and with potentially better outcomes.
What evidence suggests that this trial's treatments could be effective for neuroblastoma and other GD2-expressing cancers?
Earlier research showed that specially engineered T cells targeting GD2 held promise for treating neuroblastoma, a type of cancer. Some patients even achieved complete remission, with positive results observed at 6 weeks, 3 months, and 6 months. However, these T cells did not remain in the body long, disappearing after about 2 weeks. In this trial, participants will receive C7R-GD2.CART cells, modified by adding a new gene called C7R. This modification allows the T cells to survive longer by providing a steady supply of substances that support their survival, potentially enhancing their ability to fight cancer. GD2 is a known target in neuroblastoma, making this approach a hopeful option for patients with GD2-positive cancers.34567
Who Is on the Research Team?
Bilal Omer, MD
Principal Investigator
Baylor College of Medicine
Are You a Good Fit for This Trial?
This trial is for people aged 1-75 with certain types of cancer (like neuroblastoma, sarcoma, uveal melanoma, breast cancer) that have a substance called GD2 on their cells. The cancers should be resistant to standard treatments or have come back after treatment. Participants need to be relatively healthy otherwise, with a life expectancy of at least 12 weeks and stable organ function.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Lymphodepletion Chemotherapy
Patients may receive cyclophosphamide and fludarabine to prepare the body for T cell infusion
T Cell Infusion
Patients receive GD2-C7R T cells infusion, potentially followed by a second infusion one week later
Initial Follow-up
Patients are monitored for safety and effectiveness with follow-up visits at weeks 1, 2, 4, 6, and 8
Long-term Follow-up
Patients are monitored for long-term side effects and effectiveness of the gene transfer for up to 15 years
What Are the Treatments Tested in This Trial?
Interventions
- C7R-GD2.CART cells
Find a Clinic Near You
Who Is Running the Clinical Trial?
Baylor College of Medicine
Lead Sponsor
Center for Cell and Gene Therapy, Baylor College of Medicine
Collaborator
Cancer Prevention Research Institute of Texas
Collaborator
The Methodist Hospital Research Institute
Collaborator