On Aug. 30, 2017, the U.S. Food and Drug Administration (FDA) approved a personalized cellular therapy developed by the University of Pennsylvania and Children’s Hospital of Philadelphia (CHOP) for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. The approval was granted to Novartis for the chimeric antigen receptor (CAR) T-cell therapy, Kymriah™ (tisagenlecleucel, formerly CTL019). In 2012, Penn and Novartis entered into a global collaboration to further research, develop and commercialize Kymriah and other CAR-T cell therapies for the treatment of cancers. Kymriah is the first therapy based on gene transfer approved by the FDA.

Investigators at Penn’s Perelman School of Medicine and CHOP, who together led research, development, and clinical trials of the new therapy in collaboration with Novartis, hailed the FDA’s approval as a game changer for the treatment of younger patients battling the aggressive blood cancer and a pivotal milestone in this new era of cellular therapies that treat cancer with a patient’s own immune system.

Kymriah is expected to be available through a network of certified treatment centers throughout the United States.

The new treatment modifies patients’ own immune T cells, which are collected and reprogrammed at the Novartis manufacturing facility to potentially seek and destroy the patients’ leukemia cells. After being infused back into patients’ bodies, these newly built “hunter” cells both multiply and attack, targeting cells that express a protein called CD19. Tests reveal that the army of hunter cells can grow to more than 10,000 new cells for each single engineered cell patients receive, producing high remission rates in completely refractory leukemia—and can survive in the body for years.

CTL019 was first tested at Penn in 2010, in adult patients with advanced chronic lymphocytic leukemia (CLL). In 2012, CHOP became the first institution to investigate Kymriah in pediatric patients with ALL, the most common childhood cancer. About 20 percent of the 3,500 pediatric and young adult patients diagnosed every year with ALL in the United States relapse or do not respond to conventional treatment.