Pediatric oncologist Dr. Stephan A. Grupp of director of Translational Research for the Center for Childhood Cancer Research at The Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania said the team reprogrammed the girl's own immune cells to attack an aggressive form of childhood leukemia.
Grupp's research builds on his ongoing collaboration with University of Pennsylvania scientists who originally developed the modified T-cells as a treatment for B-cell leukemias.
By using the CTL019 treatment -- a drug -- in his pediatric patient, Grupp found the very activity that destroyed leukemia cells also stimulated a highly activated immune response called a cytokine release syndrome. The child became very ill and had to be admitted to the intensive care unit.
Grupp and his team counteracted these toxic side effects by using two immunomodulating drugs that blunted the overactive immune response and rapidly relieved the child's treatment-related symptoms.
The drugs did not interfere with the CTL019 therapy's anti-leukemia benefits, which persisted six months after the infusion of cell therapy. This persistence is essential, because the engineered T-cells remain in the patient's body to protect against a recurrence of the cancer.
"These engineered T-cells have proven to be active in B-cell leukemia in adults," Grupp said in a statement. "Our hope is that these results will lead to widely available treatments for high-risk B-cell leukemia and lymphoma, and perhaps other cancers in the future."
The findings were presented at the American Society of Hematology annual meeting in Atlanta.