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Baylor College of Medicine News

Phase 1 trial uses enhanced T cells to treat neuroblastoma

A phase 1 clinical trial at Baylor College of Medicine and Texas Children’s Cancer Center using an immunotherapy approach to treating neuroblastoma has received support from the nonprofit organization Solving Kids’ Cancer.

The organization provided $153,000 for the trial, which will start this summer. Dr. Chrystal Louis, assistant professor of pediatrics - hematology/oncology at Baylor College of Medicine, serves as principal investigator.

"Neuroblastoma is one of the most common malignant solid tumors of childhood. Since children with high-risk disease continue to have poor outcomes despite intensive therapy, new treatment strategies are required. Researchers have shown that some patients with neuroblastoma can benefit from immunotherapy," said Louis, who is also with the Center for Cell and Gene Therapy at BCM, Texas Children’s Hospital and the Methodist Hospital.

The disease is susceptible to antibodies that target the GD2 antigen expressed by the tumor and to T cell immune responses triggered by tumor vaccines. Louis and her research colleagues have worked on a chimeric antigen receptor (CAR) that when placed on T cells combines the ability to recognize the GD2 antigen on tumor cells with the capacity to signal the T cells to kill the tumor cells.

Placing these CARs into T cells allows them to target and kill GD2-positive tumors like neuroblastoma, Louis explained. The CARs were further modified so they can make T cells more efficient and last in the body longer. To make sure the enhanced T cells are safe, researchers added a safety switch that can be triggered by an injection of a drug that is harmless to normal cells but kills more than 90 percent of cells with the enhanced safety features.

"In this clinical trial, we are testing the safety and anti-tumor activity of our enhanced GD2-CAR T cells. Patients that have no evidence of toxicity after the T cell infusion, may be eligible to receive an additional dose of T cells. Patients who experience toxicity thought to be caused by the T cells will be given a dose of medication that activates the safety switch and kills the modified T cells. We believe that safely improving the activity and longevity of GD2-CAR T cells will cause increased tumor killing and thus improved patient survival for children with this disease. The funding provided by Solving Kids’ Cancer has been crucial in allowing our research team to offer this novel clinical trial to patients with relapsed or refractory neuroblastoma," Louis said.

"Immunotherapy using modified T cells has successfully treated several types of cancer, including leukemia in children," said Scott Kennedy, the executive director of Solving Kids’ Cancer. “This phase 1 clinical trial will allow children with neuroblastoma to benefit from the same cutting-edge research and offer them a promising new treatment option.”

Solving Kids’ Cancer is a non-profit organization dedicated to improving survival of the deadliest pediatric cancers through novel clinical research. SKC finds, funds, and manages new clinical trials with the goal of introducing novel therapies that are more effective and less toxic than the current standard of care.

Texas Children’s Cancer Center is a joint program of Baylor College of Medicine and Texas Children’s Hospital.