As reported in PR Newswire, Children’s Hospital of Philadelphia (CHOP) investigators presented updated efficacy and safety data on Kymriah (tisagenlecleucel)—the first ever U.S FDA approved personalized CAR T-cell gene immunotherapy for aggressive blood cancers at the 60th American Society of Hematology annual meeting as well as first of kind research on overcoming CART T-cell resistance.
Before we proceed with the conference summary a brief digression. Kymriah is a treatment for B-cell acute lymphoblastic leukemia (ALL) which exploits the body’s own T cells to fight cancer (adoptive cell transfer). T cells from a person with cancer are removed, genetically engineered to make a specific chimeric cell surface receptor with components from both T-cell receptor and an antibody specific to a protein on the cancer cell and transferred back to the person. The T cells are engineered to target a protein called CD19 that is common on B cells. A chimeric T cell receptor (CAR-T) is expressed on the surface of the T cell.
Originally developed a team headed by the University of Pennsylvania’s Carl H June the treatment was licensed to Novartis and in April 2017 received a breakthrough therapy designation by the FDA for the treatment of relapsed or refractory diffuse large B-cell lymphoma. In August 2017 the FDA approved the use of the treatment in patients with acute lymphoblastic leukemia.
Back to the CHOP story. The ELIANA trial is the first pediatric global CAR-T cell therapy registration trial of Kymriah in children and young adult patients with relapsed or refractory (r/r) acute lymphoblastic leukemia (ALL). CHOP research updates show an 82% remission rate within three months after a single infusion, and 62% relapse-free survival at 24 months. To date no new safety concerns have emerged.