18 Participants Needed

GPC2-CAR T Cell Therapy for Brain Cancer

MD
Overseen ByMariah Duncan
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if GPC2-CAR T cell therapy is safe and effective for children and young adults with brain cancer that hasn't responded to other treatments. The therapy uses modified immune cells to target and attack cancer cells in the brain. Individuals diagnosed with specific types of brain tumors, such as medulloblastoma, whose cancer has returned or not responded to other therapies, might be suitable candidates for this trial. It is particularly for those who have tried other treatments without success and seek new options to control their disease. As a Phase 1 trial, the research focuses on understanding how the treatment works in participants, offering them the opportunity to be among the first to receive this innovative therapy.

Do I have to stop taking my current medications for the trial?

The trial requires a washout period (time without taking certain medications) for some treatments. You must wait at least 21 days or 5 half-lives after any prior systemic therapy, 28 days after bevacizumab, and 30 days after any investigational drug. Check with the trial team about your specific medications.

Is there any evidence suggesting that GPC2-CAR T cell therapy is likely to be safe for humans?

Research has shown that GPC2-CAR T cells have promising results in early studies. These studies indicate that these cells can effectively locate and attack cancer cells, particularly in cancers like neuroblastoma, which begins in nerve tissue. However, questions remain about how well individuals can tolerate these treatments.

In lab tests, GPC2-CAR T cells demonstrated a strong ability to fight tumors and remain active in the body for a significant period, which is beneficial for combating cancer. Since this trial is just beginning, the complete safety profile for humans is not yet known.

As this treatment is in the early trial phase, researchers are primarily focused on understanding its safety and potential side effects. This phase typically involves a small group of participants to gather safety information before testing on larger groups. While early results are promising, ongoing studies are crucial to determine the treatment's safety for patients.12345

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for brain cancer, such as surgery, radiation, and chemotherapy, GPC2-CAR T cell therapy uses a cutting-edge approach that harnesses the body's own immune system to fight cancer. This treatment works by genetically engineering a patient’s T cells to specifically target the GPC2 protein found on brain cancer cells. Researchers are excited because this method has the potential to directly attack cancer cells with precision and reduce damage to healthy brain tissue. Plus, the use of intracerebroventricular delivery allows these engineered cells to reach the brain more effectively, which could improve outcomes compared to traditional therapies.

What evidence suggests that GPC2-CAR T cells might be an effective treatment for brain cancer?

Research has shown that GPC2-CAR T cells have produced promising results in early lab studies for treating brain tumors like medulloblastoma. These specially designed cells target Glypican-2 (GPC2), a protein often found in these tumors. Initial studies suggest that these cells can effectively attack tumor cells, particularly when traditional treatments have failed. Although human studies have provided limited information so far, the mechanism of these cells suggests they could help combat cancer cells in the brain. This trial will further investigate the effectiveness of GPC2-CAR T cells in real-world situations. More studies are needed to confirm these findings.12567

Who Is on the Research Team?

KR

Katherine Ryan, DO

Principal Investigator

Stanford University

Are You a Good Fit for This Trial?

This trial is for children and young adults with relapsed or refractory medulloblastoma or other CNS embryonal tumors. Participants must meet specific health criteria to be eligible, but the exact inclusion details are not provided here.

Inclusion Criteria

I've had multiple treatments and have stable or no current side effects.
I am mostly able to care for myself, but may need occasional help.
My organs and bone marrow are functioning normally.
See 9 more

Exclusion Criteria

Known sensitivity or allergy to any agents/reagents used in this study
I haven't had serious heart problems in the last year.
I do not have any severe illnesses that make treatment risky for me.
See 10 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Leukapheresis and Manufacturing

Participants undergo leukapheresis for collection of peripheral blood mononuclear cells to manufacture GPC2-CAR T cells

Up to 6 months

Lymphodepleting Chemotherapy

Participants receive lymphodepleting chemotherapy with fludarabine and cyclophosphamide

1 week

Treatment

Participants receive up to 8 cycles of intracerebroventricular GPC2-CAR T cell infusions

Up to 8 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 2 years

What Are the Treatments Tested in This Trial?

Interventions

  • GPC2-CAR T cells
Trial Overview The trial is testing GPC2-CAR T cell therapy administered directly into the cerebrospinal fluid. It's an early-phase study to check if this treatment is feasible, safe, and shows any signs of working against these brain cancers.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: GPC2-CAR T Cell TherapyExperimental Treatment3 Interventions

Find a Clinic Near You

Who Is Running the Clinical Trial?

Stanford University

Lead Sponsor

Trials
2,527
Recruited
17,430,000+

Citations

GPC2-targeted CAR T cells engineered with NFAT ...Chimeric antigen receptor (CAR) T cells have shown limited efficacy in NB, with the best outcomes reported in patients with a low tumor burden, highlighting the ...
GPC2-CAR T cells have potent preclinical activity against ...Patients with group 3 and 4 MB have poor clinical outcomes, underscoring the urgent need for new therapies. Glypican-2 (GPC2) is a recently discovered oncofetal ...
Enhanced anti-tumor activity mediated by combination ...Chimeric antigen receptor T (CAR-T) cells targeting single antigens show limited activity against solid tumors due to poor T cell persistence, low efficiency ...
Development of GPC2-directed chimeric antigen receptors ...This work aimed to characterize GPC2 expression in pediatric brain tumors and develop an mRNA CAR T cell approach against this target.
5.pubmed.ncbi.nlm.nih.govpubmed.ncbi.nlm.nih.gov/40569285/
GPC2-Targeted CAR T Cells Engineered with NFAT-Inducible ...Chimeric antigen receptor (CAR) T cells have shown limited efficacy in neuroblastoma, with the best outcomes reported in patients with a low ...
CAR T-cell-mediated delivery of bispecific innate immune ...We previously showed that GPC2.CAR T-cells have potent antitumor effects in neuroblastoma preclinical models; however, a subset of tumors ...
GPC2-CAR T cells tuned for low antigen density mediate ...GPC2-CAR T cells exhibited markedly increased anti-tumor activity, CAR expansion, and persistence (Figures 6E–6G) and also significantly ...
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