19 Participants Needed

EBV-specific CTLs for Neuroblastoma

(NESTLES Trial)

Age: < 65
Sex: Any
Trial Phase: Phase 1
Sponsor: Baylor College of Medicine
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

Trial Summary

What is the purpose of this trial?

Patients have high-risk neuroblastoma, a form of cancer typically found in children. The patients previously participated in a gene transfer research study using special immune cells. This research study combines two different ways of fighting disease, antibodies and T cells. Antibodies are types of proteins that protect the body from bacterial and other infections. T cells, also called cytotoxic T lymphocytes or CTLs, are special infection-fighting blood cells that can kill some tumor cells. Both antibodies and T cells have been used to treat patients with cancers and while they have shown promise, they have not been strong enough to cure most patients. The antibody used in this study is called 14g2a. This antibody sticks to neuroblastoma cells because of a substance on the outside of these cells called GD2. 14g2a and other antibodies that bind to GD2 have been used to treat people with neuroblastoma. For this study 14g2a has been changed so that instead of floating free in the blood, it is now joined to T cells. When an antibody is joined to a T cell in this way it is called a chimeric receptor. T lymphocytes or CTLs can kill tumor cells but there normally are not enough of them to kill all tumor cells. Some researchers have taken T cells from a person's blood, grown more of them in the laboratory and then given them back to the patient. Sometimes an antibody or chimeric receptor is attached to these T cells to help them bind to tumor cells. These chimeric receptor-T cells seem to kill some of the tumor, but they don't last very long in the body and so the tumor eventually comes back. We have found that T cells that are also trained to recognize the virus that causes infectious mononucleosis, Epstein Barr Virus (EBV), can stay in the blood stream for many years. By joining the 14g2a antibody to the CTLs that recognize EBV, we believe we will make a cell that can last a long time in the body (because they are EBV-specific) and recognize and kill neuroblastoma cells (because an antibody that can recognize these cells has been placed on their surface). Patients received treatment with the immune cells described above. They may want to receive an additional dose of these cells. This is being offered as an option because their neuroblastoma has returned and they have enough cells remaining to provide the patients with an additional dose. These 14g2a antibody CTLs are an investigational product not approved by the Food and Drug Administration.

Will I have to stop taking my current medications?

The trial protocol does not specify whether you need to stop taking your current medications. However, you must not be receiving any investigational agents or have received any tumor vaccines within the previous 6 weeks.

What data supports the effectiveness of the treatment EBV-specific CTLs for Neuroblastoma?

Research shows that GD2-directed chimeric antigen receptor (CAR) T cells, which are part of the treatment, helped shrink tumors in children with neuroblastoma. Additionally, EBV-specific CTLs expressing a GD2-specific receptor survived longer and were associated with tumor regression in some patients.12345

Is the treatment with EBV-specific CTLs for neuroblastoma safe?

Research shows that using EBV-specific CTLs for neuroblastoma appears to be safe, with some patients experiencing tumor regression or necrosis. In early clinical trials, CAR T cell therapy, which includes EBV-specific CTLs, has been found to be safe and feasible, although challenges remain in improving its effectiveness.12367

How is the EBV-specific CTLs treatment for neuroblastoma different from other treatments?

This treatment is unique because it uses genetically engineered T cells that target both the Epstein-Barr virus (EBV) and the GD2 antigen on neuroblastoma cells, enhancing their survival and antitumor activity compared to other T cells. This dual targeting approach allows the modified T cells to persist longer in the body, potentially leading to better outcomes in shrinking tumors.13458

Research Team

AA

Andras A Heczey, MD

Principal Investigator

Baylor College of Medicine

Eligibility Criteria

This trial is for children with high-risk neuroblastoma who have relapsed or persistent disease after initial therapy. They must not require oxygen (>90% on room air), expect to live at least 12 weeks, and recovered from previous chemotherapy effects. No investigational agents or tumor vaccines should have been received in the last 6 weeks. Participants need normal blood counts, liver and kidney function, no severe heart/lung issues, a decent performance score (Karnofsky/Lansky >60%), and specific immune cells from a prior study.

Inclusion Criteria

My neuroblastoma is high risk and has either come back or didn't fully respond to initial treatment.
Patients must have AST less than 5 times the upper limit of normal
Patient may not have an oxygen requirement as defined by pulse oximetry of > 90% on room air
See 16 more

Exclusion Criteria

Patients not meeting eligibility criteria

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks
1 visit (in-person)

Treatment

Participants receive escalating doses of 14g2a.zeta chimeric receptor transduced autologous EBV specific cytotoxic T-lymphocytes and 14g2a.zeta transduced autologous peripheral blood T-cells

6 weeks
Weekly visits for infusions

Follow-up

Participants are monitored for safety and effectiveness after treatment, including long-term monitoring for gene transfer side effects

15 years
Regular visits, frequency decreasing over time

Optional Additional Dose

Participants may receive an additional dose of cells if desired due to neuroblastoma recurrence

Treatment Details

Interventions

  • EBV Specific CTLs
  • GD-2 Specific Chimeric T Cell Receptors
Trial Overview The trial tests modified immune cells (CTLs) that are trained to fight both neuroblastoma cancer cells and EBV virus by using an antibody called 14g2a attached to them. These 'chimeric receptor-T cells' aim to last longer in the body and effectively target cancer cells. Patients may receive an additional dose of these CTLs if their cancer has returned.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: EBV specific CTLs w/out lymphodepletionExperimental Treatment1 Intervention
Escalating doses of 14g2a.zeta chimeric receptor transduced autologous EBV specific cytotoxic T-lymphocytes (EBV-CTL) and 14g2a.zeta transduced autologous peripheral blood T-cells administered to patients with Neuroblastoma.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Baylor College of Medicine

Lead Sponsor

Trials
1,044
Recruited
6,031,000+

Center for Cell and Gene Therapy, Baylor College of Medicine

Collaborator

Trials
114
Recruited
2,900+

Findings from Research

In a study involving 19 patients with high-risk neuroblastoma, GD2 chimeric antigen receptor (CAR) T cells were able to induce complete remission in 3 out of 11 patients with active disease, demonstrating their efficacy in treating this aggressive cancer.
The persistence of CAR T cells in the patients was linked to better clinical outcomes, with some CAR-ATCs lasting up to 192 weeks, suggesting that longer-lasting CAR T cells may contribute to improved survival rates.
Antitumor activity and long-term fate of chimeric antigen receptor-positive T cells in patients with neuroblastoma.Louis, CU., Savoldo, B., Dotti, G., et al.[2023]
Despite the integration of anti-GD2 monoclonal antibody therapy, the 5-year overall survival rate for high-risk neuroblastoma patients remains around 50%, highlighting the need for improved treatment options.
CAR T cell therapy has shown promise in early clinical trials for neuroblastoma, demonstrating safety and feasibility, but challenges such as T cell persistence and an immunosuppressive tumor environment need to be addressed to enhance its efficacy.
CAR T Cell Therapy for Neuroblastoma.Richards, RM., Sotillo, E., Majzner, RG.[2023]
A 3rd generation CAR T-cell construct targeting GD2 showed improved anti-tumor efficacy in neuroblastoma compared to other constructs, highlighting the importance of the 4-1BB costimulatory domain in enhancing T-cell characteristics like persistence and tumor control.
The inclusion of an inducible safety switch (iC9) in the CAR construct allows for the controlled elimination of CAR T cells if necessary, without compromising their anti-tumor activity, making it a safer option for treating pediatric patients.
Choice of costimulatory domains and of cytokines determines CAR T-cell activity in neuroblastoma.Quintarelli, C., Orlando, D., Boffa, I., et al.[2021]

References

Antitumor activity and long-term fate of chimeric antigen receptor-positive T cells in patients with neuroblastoma. [2023]
CAR T Cell Therapy for Neuroblastoma. [2023]
Virus-specific T cells engineered to coexpress tumor-specific receptors: persistence and antitumor activity in individuals with neuroblastoma. [2023]
Genetic engineering of cytolytic T lymphocytes for adoptive T-cell therapy of neuroblastoma. [2017]
CAR T Cells Offer Hope for Neuroblastoma. [2023]
Choice of costimulatory domains and of cytokines determines CAR T-cell activity in neuroblastoma. [2021]
Targeting GD2-positive Refractory/Resistant Neuroblastoma and Osteosarcoma with Anti- CD3 x Anti-GD2 Bispecific Antibody Armed T cells. [2023]
CAR T Cells Engineered to Target Low-Density Antigens Show Efficacy and Safety. [2022]