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


Our study evaluates the safety, persistence and antitumor activity of T cells bearing a 2nd generation GD2-specific chimeric antigen receptor (CAR) for patients with H3K27M altered diffuse midline gliomas (DMG). Starting at the second dose level, we will add an interleukin-7 receptor gene (C7R) to the CAR T cells that is constitutively active. The goal is to improve persistence of the T cells by providing cytokine support with C7R.

The two main disease groups eligible for this trial are:

  1. Patients with newly diagnosed DIPG (prior to progression only) with confirmation of positive H3K27M mutation status, including spinal glioma.
  2. Patients with newly diagnosed or relapsed HGG (before OR after progression) with confirmation of positive H3K27M mutation status, including spinal glioma.

Patients with other brain tumors, such as medulloblastoma, are also eligible if GD2 expression of tumor tissue is confirmed.


The study is conducted in two parts:
  • Prior to cell infusion:
    • Procurement. Procurement consent can be obtained remotely. White blood cells are collected by a blood draw of 60 ml. Blood can be collected remotely at the referral institution if preferred and shipped to Texas Children’s Hospital. Cell generation and testing takes at least 4-5 weeks. Procurement can be completed before, during or after radiation therapy (XRT).
  • Chemotherapy and cell infusion. Will occur at least 4 weeks after completion of standard XRT in newly diagnosed patients.
    • Ommaya placement. The patient will need to have an Ommaya in place or have Ommaya placement arranged at referral institution or by our team in Houston before treatment. The Ommaya is used for safe removal of CSF in the case increased intracranial pressure develops and not currently used for cell infusion.
    • Treatment. We will administer standard lymphodepleting chemotherapy with cyclophosphamide and fludarabine, which will be given inpatient or outpatient over 3 days, followed by the intravenous CAR T cell infusion and inpatient monitoring for at least 5 days. After that the patient will be evaluated outpatient until week 4 and is expected to stay in Houston during this time; after this period they can return home.
    • For disease assessment, scans can be done at the referring site.
  • Potential toxicity: The main anticipated toxicity is an inflammatory response at the tumor size, which could cause significant neurological toxicity and possibly cytokine release syndrome. Risk and benefits can be discussed with the principal investigators, Dr. Bilal Omer ( and Dr. Frank Lin.


Eligible disease are described above. Other key eligibility criteria, include standard organ function criteria, Lansky/Karnofsky >50, tumor size <5 cm at diagnosis, low or no steroid dose at time of treatment. All patients need to complete standard radiation therapy before treatment. Complete eligibility criteria can be reviewed at Scans will be reviewed by the investigators before final eligibility determination.