What side effects are associated with this type of intervention?

Common treatments for Diffuse Intrinsic Pontine Glioma (DIPG) include radiation therapy, chemotherapy, and experimental therapies such as CAR T cell therapy. Radiation therapy can cause side effects like fatigue, hair loss, and skin changes. Chemotherapy side effects vary depending on the drug but often include nausea, vomiting, and lowered blood cell counts. CAR T cell therapies, such as those targeting B7-H3, EGFR806, HER2, and IL13-zetakine, can lead to cytokine release syndrome (CRS), which includes symptoms like fever, nausea, headache, and hypotension, as well as neurotoxicity, which can manifest as confusion, seizures, or encephalopathy.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Diffuse Intrinsic Pontine Glioma (DIPG). Traditional treatment approaches have included radiation therapy and, in some cases, chemotherapy, but these have not significantly improved long-term survival. The SC-CAR4BRAIN trial is investigating a novel CAR T cell therapy targeting B7-H3, EGFR806, HER2, and IL13-zetakine, which represents an innovative approach in the treatment of DIPG. While CAR T cell therapies have been FDA-approved for certain hematologic malignancies, their application in solid tumors like DIPG is still under clinical investigation.

What other types of research exist?

Promising novel alternatives to SC-CAR4BRAIN for DIPG patients include: 1) ONC201, a small molecule DRD2 antagonist that has shown encouraging results in clinical trials for DIPG. 2) Panobinostat, a histone deacetylase inhibitor, which has demonstrated potential in preclinical models and early-phase clinical trials. 3) GD2-targeted CAR T cell therapy, which is being explored for its efficacy in treating DIPG. These alternatives represent emerging therapeutic strategies that may offer new hope for DIPG patients.

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CAR T-cell Therapy

CAR T Cell Therapy for Pediatric Brain Cancer

Phase 1

Recruiting

Led By Rebecca Ronsley, MD

Research Sponsored by Seattle Children's Hospital

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 28 days post-final sc-car4brain infusion

Awards & highlights

No Placebo-Only Group

Summary

This trial uses a patient's own modified immune cells to treat aggressive brain tumors in children and young adults. The immune cells are enhanced to better recognize and attack cancer cells in the brain. This approach has shown remarkable results in treating certain cancers and is now being explored for brain tumors.

Who is the study for?

This trial is for children and young adults with specific brain tumors like DIPG, DMG, or recurrent/refractory CNS tumors. They must have a life expectancy of at least 8 weeks, be in good physical condition (Lansky/Karnofsky score ≥ 60), have proper organ function, agree to use contraception if applicable, and have a catheter placed for treatment delivery.

What is being tested?

The study tests SC-CAR4BRAIN therapy using the patient's own T cells engineered to target tumor cells via CARs that recognize B7-H3, EGFR806, HER2, and IL13-zetakine. The modified T cells are delivered directly into the brain through a catheter. Patients are divided into two groups based on their type of tumor.

What are the potential side effects?

Potential side effects may include reactions related to immune response such as inflammation in various organs or tissues due to targeted cell destruction by CAR T-cells; infusion-related reactions; fatigue; possible neurological symptoms from direct brain administration.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 28 days post-final sc-car4brain infusion

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~28 days post-final sc-car4brain infusion

This trial's timeline: 3 weeks for screening, Varies for treatment, and 28 days post-final sc-car4brain infusion for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Administration feasibility

Dose level

Manufacturing Feasibility

+1 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Arm B - DMG & recurrent/refractory tumorsExperimental Treatment1 Intervention

Group II: Arm A - DIPGExperimental Treatment1 Intervention

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatment for Diffuse Intrinsic Pontine Glioma (DIPG) is radiation therapy, which works by damaging the DNA of cancer cells, thereby inhibiting their ability to replicate and grow. However, this treatment only temporarily alters the clinical course of the disease. Emerging treatments like CAR T-cell therapy, as studied in the SC-CAR4BRAIN trial, involve engineering a patient's T cells to express chimeric antigen receptors (CARs) that specifically target tumor-associated antigens such as B7-H3, EGFR806, HER2, and IL13-zetakine. These CAR T cells can recognize and kill tumor cells more effectively. This targeted approach is crucial for DIPG patients because it offers a potential for more precise and effective treatment, potentially leading to better outcomes in a disease that is otherwise difficult to treat.

Safety and Efficacy of Chimeric Antigen Receptor T-Cell Therapy for Glioblastoma: A Systemic Review and Meta-Analysis.