Texas Children's Cancer and Hematology Center 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) augmented by a constitutively active interleukin-7 receptor gene (C7R) to the CAR T cells for treatment of  patients with H3K27M-altered diffuse midline gliomas (DMG) and other recurrent high grade CNS tumors expressing GD2. The goal is to improve persistence of the CAR T cells using C7R.

The three main disease groups eligible for this trial are:

  1. Patients with newly diagnosed pontine DMG (DIPG, prior to progression only) with confirmation of tumor H3K27-alteration.
  2. Patients with newly diagnosed or relapsed DMG located away from the pons (before OR after progression) with confirmation of H3K27-alteration status, including spinal glioma.
  3. Patients with other recurrent or progressive high grade central nervous system (CNS) tumors (such as medulloblastoma) that test positive for GD2.


This 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 70 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 4-6 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 the referral institution or by our team in Houston before treatment. The Ommaya is used for cell infusion and as a safety mechanism (for removal of CSF in the case increased intracranial pressure develops).
    • Treatment. We will administer standard lymphodepleting chemotherapy with cyclophosphamide and fludarabine, which will be given inpatient over 3 days. CAR T cells are then administered intraventricularly through the Ommaya, followed by two intravenous infusions 5 days apart. CAR T cells are administered as an inpatient, allowing for close observation. After discharge, the patient will be evaluated in the outpatient clinic until week 4 and is expected to stay in Houston during this time; after this period they can return home.
    • For disease assessment, MRIs can be done at the referring site.
  • Potential toxicity: The main anticipated toxicity is an inflammatory response at the primary tumor site, which could cause significant neurological toxicity, and possibly cytokine release syndrome.

Risks and benefits can be discussed with the principal investigators, Drs. Bilal Omer, Frank Lin and Austin Stuckert.


Eligible diseases 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 clinicaltrials.gov. Scans will be reviewed by the investigators before final eligibility determination.

Stacey L. Berg, MD

Stacey Berg, MD

Texas Children’s Cancer and Hematology Centers