Study of GD2-Specific CAR T Cell Therapy with IL-7 Receptor Gene to Treat GD2-Expressing Brain Tumors in Children and Young Adults

What this Trial Is About

Researchers are evaluating a gene therapy approach using specially modified T cells called GD2-C7R CAR T cells for treating children and young adults aged 1 to 25 years with certain GD2-expressing brain tumors. These include diffuse midline glioma, diffuse intrinsic pontine glioma, medulloblastoma, and other rare high-grade gliomas that currently have no standard effective treatment. The study aims to find the largest safe dose of these modified T cells delivered both intravenously and directly into the brains fluid spaces. Participants first have their blood collected to prepare the GD2-C7R CAR T cells by inserting genes that help the T cells recognize and kill cancer cells and survive longer. The initial treatment cycle includes an intravenous infusion of these cells combined with chemotherapy to help the T cells grow. Subsequent treatment cycles involve infusions directly into the brain through an Ommaya reservoir or programmable VP shunt. Patients receive up to 24 treatment cycles spaced at least 4 weeks apart, with careful dose adjustments and hospital monitoring. Throughout the study, participants undergo regular physical exams, blood tests, MRI scans, spinal fluid tests, and neurological evaluations to monitor tumor response and treatment safety. Blood and spinal fluid samples are collected at multiple timepoints up to 15 years after treatment to study the persistence and effects of the T cells. Hospital stays for monitoring side effects last several days after each infusion, with close follow-up visits scheduled frequently during the first year and then annually for long-term safety and effectiveness assessment.

C7R-GD2.CAR T Cells for Patients With GD2-expressing Brain Tumors (GAIL-B)