Penn to Broaden Pathways for Development of New CAR T Cell Therapies

Following a landmark, first-of-its-kind collaboration that spawned the global CAR T cell therapy industry, the University of Pennsylvania and Novartis have concluded their seven-year research and development alliance, which resulted in the world’s first approved gene therapy for cancer. They will continue to work together on a more narrow CAR T cell therapy clinical trials portfolio.

Penn and Novartis joined forces in 2012, a year after a team of physicians and scientists from Penn’s Abramson Cancer Center published breakthrough results in several chronic lymphocytic leukemia patients treated with personalized versions of their own immune cells. The exclusive agreement, in collaboration with Children’s Hospital of Philadelphia, led to the August 2017 U.S. Food and Drug Administration approval of the CAR-modified T cell therapy Kymriah® for the treatment of pediatric and young adult acute lymphoblastic leukemia. In May 2018, the therapy received a second approval, for the treatment of non-Hodgkin lymphoma. International approvals have stretched from Canada to Europe to Japan.

“Our highly successful collaboration with Novartis has been an inspiring example of how experts from academic medicine can unite with industry to further develop and bring discoveries from the laboratory to patients across the world who are in desperate need of new treatment options,” said J. Larry Jameson, MD, PhD, Executive Vice President of the University of Pennsylvania for the Health System and dean of the Perelman School of Medicine. “The FDA approval for Kymriah came in record time, and we are committed to continuing the momentum as we develop new approaches to expand CAR T cell therapies into more cancers, and beyond.”

Penn’s CAR T cell research team is led by Carl June, MD, the Richard W. Vague Professor in Immunotherapy and director of the Center for Cellular Immunotherapies.

A new agreement between Penn and Novartis ends the broad alliance between them while ensuring continued collaboration around more narrowly focused projects, including four clinical trials. They include a multiple myeloma trial involving two different CAR T cell therapies and a trial harnessing CAR T cells in tandem with pembrolizumab for the deadly brain cancer glioblastoma, and two new CAR T cell trials – targeting CD22 and CD123 – for blood cancers.

“In Philadelphia alone, there are 30 new companies with 3,000 new jobs representing $2 billion of investment in cell and gene therapy — what we call the Cellicon Valley,” said Robert H. Vonderheide, MD, DPhil, director of the Abramson Cancer Center of the University of Pennsylvania. “As Penn scientists continue to accelerate innovation in T cell therapies, collaborations beyond one company are critical for us to advance these ideas and bring new options and hope to more patients.”

The Penn team will continue to advance its translational and clinical research portfolios in the CAR T cell space, including development of next-generation CAR T treatments in both solid tumors and blood cancers. Additional priorities include improving manufacturing processes for these personalized therapies, as well as development of novel clinical trial designs. The Penn researchers are also at work on efforts to improve the function of existing CAR T cell products, and to develop multi-modal therapies to enhance efficacy or reduce side effects of the treatment.

Editor’s Note: The University of Pennsylvania has licensed some technologies associated with certain CAR T cell therapy products to Novartis. Some of the scientists involved in these trials are inventors of these technologies. As a result of the continued licensing relationship with Novartis with respect to these technologies, the University of Pennsylvania continues to receive significant financial benefit, and some of these inventors have benefitted financially and/or may benefit financially in the future.