FDA Grants Breakthrough Designation to Personalized Immunotherapy for ALL

“The FDA today granted breakthrough therapy designation for CTL019, an investigational personalized immunotherapy, for the treatment of relapsed and refractory adult and pediatric acute lymphoblastic leukemia, according to a press release issued by Penn Medicine.

“CTL019 (Novartis), developed by the University of Pennsylvania, is the first personalized cellular therapy for the treatment of cancer to receive this classification.

“In early-stage clinical trials conducted at the Hospital of the University of Pennsylvania and Children’s Hospital of Philadelphia, 89% of patients with ALL who were not responding to conventional therapies achieved complete remission after treatment with 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, personalized immunotherapy,’ Carl H. June, MD, director of translational research in the Abramson Cancer Center of the University of Pennsylvania. ‘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.’ ”

Editor’s note: This story describes a new leukemia treatment called CTL019, which boosts a patient’s own immune system to fight cancer. CTL019 treatment is personalized for each patient, since it involves altering a patient’s immune system cells to attack tumor cells. It has been tested in volunteer patients in clinical trials, and has shown promising results for adults and children with relapsed or refractory acute lymphoblastic leukemia (ALL). The U.S. Food and Drug Administration (FDA) has now granted breakthrough therapy designation for CTL019, meaning that review and approval will be accelerated so that the drug can more quickly reach patients outside of clinical trials.