A University of Pennsylvania-developed personalised immunotherapy has been awarded the FDA's breakthrough therapy designation for the treatment of relapsed and refractory adult and pediatric acute lymphoblastic leukemia (ALL). The investigational therapy, known as CTL019, is the first personalised cellular therapy for the treatment of cancer to receive this important classification.
In early-stage clinical trials at the Hospital of the University of Pennsylvania and the Children's Hospital of Philadelphia, 89% of ALL patients who were not responding to conventional therapies went into complete remission after receiving CTL019.
"Our early findings reveal tremendous promise for a desperate group of patients, many of whom have been able to return to their normal lives at school and work after receiving this new, personalised immunotherapy," said the Penn research team's leader, Carl June, MD. "Receiving the FDA's breakthrough designation is an essential step in our work with Novartis to expand this therapy to patients across the world who desperately need new options to help them fight this disease."
In August 2012, Penn announced an exclusive global research and licensing agreement with Novartis to further study, develop and commercialise personalised chimeric antigen receptor (CAR) T cell therapies for the treatment of cancers. Trials employing CTL019 began in the summer of 2010 in patients with relapsed and refractory chronic lymphocytic leukemia (CLL), and are now underway for adult and pediatric patients with ALL, and patients with non-Hodgkin lymphoma and myeloma. Penn and Novartis are also investigating the next generation of CAR therapies, with trials for mesothelioma, ovarian, breast and pancreatic cancer now in early stages.
During the 2013 annual meeting of the American Society of Hematology, the Penn research team announced study results of the first 27 ALL patients (22 children and five adults) treated with CTL019: 89% of the patients had a complete response to the therapy. The first pediatric ALL patient to receive the Penn therapy celebrated the second anniversary of her cancer remission in May, and the first adult patient remains in remission one year after receiving the therapy.
The investigational treatment pioneered by the Penn team begins by removing patients' T cells via an apheresis process similar to blood donation, then genetically reprogramming them in Penn's Clinical Cell and Vaccine Production Facility. After being infused back into patients' bodies, these newly built "hunter" cells both multiply and attack, targeting tumor 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.