Immunotherapy Penn Receives FDA breakthrough therapy designation for ALL treatment

Immunotherapy Penn Receives FDA breakthrough therapy designation for ALL treatment -

At the University of Pennsylvania developed a personalized immunotherapy has been awarded the designation of breakthrough therapy from the US Food and Drug Administration for the treatment of relapsed and refractory adult and pediatric acute lymphoblastic leukemia (ALL). The experimental treatment, known as CTL019 is the first personalized cell therapy for the treatment of cancer to receive this important classification.

In clinical trials at an early stage of the hospital of the University of Pennsylvania and Children's Hospital of Philadelphia, 89 percent of all patients who did not respond to conventional treatments in complete remission after receiving CTL019.

"Our early results show great promise for a desperate group of patients, many of whom were able to return to their normal school life and work after receiving this new, personalized immunotherapy" said the head of the Penn research team Carl June, MD , Professor Richard W. Vague in immunotherapy in the department of pathology and laboratory Medicine at the Perelman school of Medicine and Director of translational research in the Abramson Cancer Center of the University of Pennsylvania. "Receiving FDA Designation breakthrough is a key step in our work with Novartis to develop this therapy to patients throughout the world who desperately need new options for help fight against this disease. "

breakthrough therapy designation from the FDA, created in 2012, aims to accelerate the development and review of new drugs - drugs and biological agents - that treat serious or life-threatening conditions, if the therapy demonstrated a substantial improvement over available treatments. The FDA has already granted Therapy Breakthrough only four other biological agents.

In August 2012, Penn announced an agreement for research and exclusive worldwide agreement with Novartis to study, develop and market custom chimeric antigen receptor (CAR) T cell therapies for the treatment of cancers. Tests with CTL019 began in summer 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 myeloma. Penn and Novartis are also working on the next generation of CAR therapies, with trials for mesothelioma, ovarian, breast and pancreatic cancer at an early stage now.

During the 2013 annual meeting of the American Society of Hematology, the Penn research team announced the results of the study of the first 27 all patients (22 children and five adults) treated with CTL019 89 percent of patients had a complete response to therapy. The first pediatric ALL patient receiving the therapy Penn celebrated the second anniversary of his remission of cancer in May, and the first adult patient remains in remission a year after receiving the therapy.

The experimental treatment developed by the Penn team begins by removing T cells from the patient through a similar process of apheresis blood donation and genetically reprogram the cell Penn clinical and vaccine production facilities. After infused back into the patients body, these new cells built "hunters" to both multiply and attack targeting tumor cells that express a protein called CD19. Tests reveal that the army of hunters cells can reach more than 10,000 new cells for each single modified cells of patients receive.

The adult CTL019 all trials at the University of Pennsylvania Abramson Cancer Center are directed by David Porter, MD , Jodi Fisher Professor Horowitz care excellence leukemia and director of the blood and marrow transplant in the Abramson Cancer Center and Noelle Frey, MD, MSCE , assistant professor of medicine at the Abramson Cancer Center. Pediatrics All tests are conducted by Stephan Grupp, MD, PhD , professor of pediatrics and director of translational research in the Center for Research on Early Childhood cancer at the Hospital of Philadelphia children . Bruce Levine, PhD , Barbara and Edward Netter Professor in cancer gene therapy in the Department of Pathology and Laboratory Medicine, is leading clinical cell Penn and vaccine production facilities.