UPenn Research Makes Landmark Strides in CAR T-Cell Cancer Immunotherapy

by Rachel DeRita, PhD candidate (Thomas Jefferson University, Department of Cancer Biology): Cancer immunotherapy is rapidly becoming one of the largest clinical and pre-clinical cancer research fields. You may even hear it talked about at your holiday cocktail party. In particular, the University of Pennsylvania has had a landmark year involving a particular type of immunotherapy, chimeric antigen receptor (CAR) T-cell therapy. This therapy involves taking a patient’s own T-cells and, outside the body, putting them through a cellular “boot camp” and training them to target a specific marker present on the cancer cell, but not normal cells. Those engineered T-cells are then administered back to the patient. Starting on August 30, 2017, the University of Pennsylvania (UPenn) and the Children’s Hospital of Philadelphia (CHOP) officially announced that the pioneering studies done at both institutions led to the first-ever cancer cell and gene therapy approval by the FDA. The approval was given to Novartis for Kymriah(TM) (formerly CTL019) to treat pediatric patients up to the age of 25 for B-cell precursor acute lymphoblastic leukemia (ALL). The T-cells in this therapy are primed and expanded to target cancer cells positive for a protein called CD19 present on the cancerous lymphocytes. Early-stage clinical trials observed more than 90% of patients achieving complete remission, leading to a global trial in 2015 that included 68 children and young adults with advanced ALL all over the world. After a single dose of their own engineered T-cells, a remarkable 83% of patients achieved complete remission. According to the investigators at UPenn’s Perelman School of Medicine and CHOP, in collaboration with Novartis, this marks a milestone for the treatment of younger patients with aggressive blood cancer.
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