Relapsed or Refractory Neuroblastoma - Healing Genes

Relapsed or Refractory Neuroblastoma

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C7R-GD2 CART Cells for Patients With Relapsed or Refractory Neuroblastoma (GAIL-N)

Phase I Study of Autologous T Lymphocytes Expressing GD2-specific Chimeric Antigen and Constitutively Active IL-7 Receptors for the Treatment of Patients With Relapsed or Refractory Neuroblastoma (GAIL-N)

Phase 1


Researchers with Texas Children’s Hospital seek patients with neuroblastoma that has either come back after treatment or did not respond to standard or other investigational treatments, to trial a gene transfer using special immune cells called T cells. In the lab, researchers put a new gene into T cells that will make them recognize GD2, a protein found on almost all neuroblastoma cells (GD2-CAR) and kill them, as well as a C7R gene to make the cells survive longer, then infuse them back to the patients.

The treatment will require blood draw to isolate the cells required, then after they are gene-modified but before they are re-infused, chemotherapy will prepare the immune system to accept them. Follow up continues for 8 weeks post therapy.


  • Be 1 to 17 years of age
  • Have a diagnosis of neuroblastoma with persistent or relapsed disease
  • Life expectancy ≥ 12 weeks


  1. Screening before the treatment (including lab tests, MRIs, physical assessments, and nerve biopsies).
  2. T-cells will be drawn and modified in the lab over several weeks.
  3. Chemotherapy will be given to prepare the immune system.
  4. Re-infusion of the modified T-cells.
  5. Follow-up for 8 weeks.

Trials will take place at the Texas Children’s Hospital in Houston, TX. Map.
Bilal Omer, MD  |  832-824-6855  |  [email protected]
Jacqueline Castello  |  832-824-4391  |  [email protected]
Baylor College of Medicine
Texas Children’s Hospital
Center for Cell and Gene Therapy, Baylor College of Medicine
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