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Engineered Immune Cells Produce Complete Response in Child with an Aggressive Pediatric Leukemia

By reprogramming a 7-year-old girl's own immune cells to attack an aggressive form of childhood leukemia, a pediatric oncologist has achieved a complete response in his patient, who faced grim prospects when she relapsed after conventional treatment. The innovative experimental therapy used bioengineered T cells, custom-designed to multiply rapidly in the patient, and then destroy leukemia cells. After the treatment, the child's doctors found that she had no evidence of cancer.

Pediatric oncologist Stephan A. Grupp, MD, PhD, of The Children's Hospital of Philadelphia, and colleagues from the University of Pennsylvania presented updated results of the clinical trial involving these engineered cells at the American Society of Hematology (ASH) annual meeting today in Atlanta. Grupp is the director of Translational Research for the Center for Childhood Cancer Research at The Children's Hospital of Philadelphia, and a professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania.

Pediatric research builds on collaboration with Penn scientists developing treatments for adult B-cell leukemias

Grupp's research builds on his ongoing collaboration with Penn scientists who originally developed the modified T cells as a treatment for B-cell leukemias. The Penn team reported on early results of a trial using this cell therapy in adult chronic lymphocytic leukemia (CLL) patients in August of 2011. Carl H. June, MD, of the Perelman School of Medicine at the University of Pennsylvania, leads this research group, which along with Grupp's work, is presenting new data at the ASH meeting showing that nine of 12 patients with advanced leukemias in the clinical trial, including two children, responded to treatment with CTL019 cells.