Teen first in Virginia to receive cancer gene therapy in UVA clinical trial

CHARLOTTESVILLE, Va. – A teenager in Virginia becomes the first in the state to receive cancer gene therapy through a University of Virginia Health System clinical trial.

The approach, known as chimeric antigen receptor (CAR) T-cell therapy, takes a person’s own immune cells and genetically modifies them with the goal of making them more effective cancer killers, says UVA Health System.

The teen was administered an experimental immunotherapy for pediatric acute lymphoblastic leukemia. The type of cancer has resisted other forms of treatment.

The experimental approach is similar to the gene therapy that the federal Food and Drug Administration approved for pediatric lymphoblastic leukemia at the end of Aug., the first gene therapy ever approved in the United States.

In this case, pediatric oncologist Dr. Daniel “Trey” Lee took the immune cells from a 14-year-old clinical trial participant with a form of cancer known as primary refractory acute lymphoblastic leukemia.

The trial participant’s immune cells were then genetically engineered by the cell therapy company Kite Pharma before Lee infused them back into the teen.

Lee hopes the new approach will offer advantages for patients.

“We collect cells from a vein of a patient, the cells are shipped to a central manufacturing facility where they’re made in approximately seven days, and they’re shipped back to us,” Lee said. “So we can have a vein-to-vein time of 16 to 18 days, which is really fast.”

While at the National Institutes of Health, Lee tested the approach using a similar product in more than 50 trial participants. Those participants saw a response rate of approximately 70 percent and a relapse rate of less than 10 percent, he said.

The results of the current testing will be available when the 20-site clinical trial has concluded, possibly in less than two years.

In addition to the leukemia therapy, Lee and his associates at UVA  are seeking to develop new T-cell therapies for deadly forms of pediatric brain cancer.

“You will see a lot more CAR T-cell treatments coming out of UVA in the future,” says Lee.

Participants of the trial must be between the ages of two and 21, and have lymphoblastic leukemia that has relapsed or not responded to other forms of treatment. There are other eligibility requirements as well.