Industry-Academic Partnership Will Establish New Research Center to Expedite Study and Development of Gene Transfer Approach

PHILADELPHIA — In an alliance aimed at bringing a new, personalized immunotherapy approach to patients with a wide variety of cancers, the University of Pennsylvania and Novartis announced today an exclusive global research and licensing agreement to further study and commercialize novel cellular immunotherapies using chimeric antigen receptor (CAR) technologies. The agreement, which follows a Penn research team's 2011 publication of breakthrough results in several chronic lymphocytic leukemia patients treated with this personalized immunotherapy technique, paves the way for pivotal studies that have the potential to expand the use of CAR therapies for additional cancers.

The new alliance represents a marquee achievement in Penn's commitment to translational science aimed at expediting the process of bringing novel therapies to patients. Together, Penn and Novartis will build a first-of-its-kind Center for Advanced Cellular Therapies (CACT) on the Penn campus in Philadelphia -- a venture which will bring full circle the 1960 discovery of the Philadelphia chromosome, the first description of a chromosome abnormality that causes cancer. The center will be devoted to the discovery, development and manufacturing of adoptive T cell immunotherapies through a joint research and development program led by scientists and clinicians from Penn, Novartis, and the Novartis Institutes for Biomedical Research.

"Penn's intellectual resources, combined with a pharmaceutical industry leader like Novartis, offer a powerful symbiotic relationship in our mutual goal of finding more effective treatments for cancer," said J. Larry Jameson, MD, PhD, dean of the Perelman School of the Medicine at the University of Pennsylvania and executive vice president for the Health System. "With our shared commitment to rapidly advancing new therapies and cures, this new alliance will provide the support for the essential clinical trials with engineered T cells, which could open doors for use of promising treatment options for many cancer patients who have reached the end of currently available treatments."

Under the terms of the agreement, Penn grants Novartis an exclusive worldwide license to the technologies used in an ongoing trial of patients with chronic lymphocytic leukemia (CLL) as well as future CAR-based therapies developed through the collaboration. Novartis will invest in the establishment of the CACT and future research of the technology. Additional milestone and royalty payments to Penn are also part of the agreement.

In August 2011, the Penn team detailed the results of an early trial utilizing the modified T cell approach among a small group of advanced chronic lymphocytic leukemia patients in the New England Journal of Medicine and Science Translational Medicine. The findings — including reports on two patients who remained in remission more than a year after their treatment — served as the first successful and sustained demonstration of the use of gene transfer therapy to create T cells aimed at battling cancerous tumors. The protocol involves removing a patient's cells and modifying them in Penn's cell and vaccine production facility, then infusing the new cells back into the patient's body following chemotherapy to attack their remaining tumors. Thus far, the study has involved only patients whose cancers have not responded to traditional therapy. These patients' only remaining treatment options would have been a bone marrow transplant, a procedure which carries a mortality risk of at least 20 percent.

"Our early results in patients treated with chimeric antigen receptors represent two decades of investment and perseverance in our effort to treat cancer in an entirely new way, combining a highly targeted cell-based therapy with the might of a patient's own immune system," said the study's leader, Carl June, MD, a professor of Pathology and Laboratory Medicine in the Perelman School of Medicine and director of Translational Research at Penn's Abramson Cancer Center. "By joining forces with Novartis, we will now have the resources and space to expand our research in new directions that we hope will change the way cancers of all kinds are treated."

Although further studies are needed to explore the long-term viability of the treatment, June's team showed that in the patients studied so far, months after infusion, the new cells had multiplied throughout the patients' bodies and were capable of continuing their "seek-and-destroy" mission against cancerous cells.

In addition to continued trials in CLL, Penn also has engineered T cell trials underway for other leukemias as well as lymphoma, mesothelioma, myeloma, and neuroblastoma. To read more about the early results of Penn's work using the new treatment approach and watch a video of June and his co-investigators discussing the research, visit Penn Medicine's online press kit.

Patients seeking information about cancer trials using CAR therapies may call the Abramson Cancer Center Clinical Trials Service at 1-855-216-0098 or visit www.pennmedicine.org/cancertrials.