Basel, Switzerland (Scicasts) – Novartis and the University of Pennsylvania (Penn) have announced an exclusive global collaboration to research, develop and commercialize targeted chimeric antigen receptor (CAR) immunotherapies for the treatment of cancers.
In addition, the parties will jointly establish a new research and development facility on the Penn campus, called the Center for Advanced Cellular Therapies (CACT).
In CAR immunotherapy, immune cells (T cells) are drawn from a patient's blood. Then, using CAR technology, the T cells are re-coded to identify and seek out cells that express proteins present on a patient's cancerous tumour. When the T cells are re-introduced into the patient's blood, they bind to the targeted cancer cells and destroy them.
As part of the transaction, Novartis acquired exclusive rights from Penn to CART-19, a novel investigational CAR therapy, currently being studied by Penn in a pilot clinical trial. CART-19 targets a protein called CD19 that is associated with a number of B-cell malignancies such as chronic lymphocytic leukaemia (CLL), B-cell acute lymphocytic leukaemia and diffuse large B-cell lymphoma.
To accelerate the discovery and development of additional therapies using CAR immunotherapy, Novartis and Penn will build the Center for Advanced Cellular Therapies on the Penn campus in Philadelphia. This will be a first-of-its-kind research and development centre established specifically to develop and manufacture adoptive T-cell immunotherapies under the research collaboration guided by scientists and clinicians from Novartis and Penn.
Early results from a clinical trial of CART-19, conducted by Penn, showed potent antileukaemic effects in three patients with advanced CLL who had previously undergone multiple courses of chemotherapy and biological therapy.
Two of the patients were still in complete remission more than a year into the CART-19 trial, and the third patient maintained partial remission for more than seven months. An immune deficiency known as hypogammaglobulinemia, an expected chronic toxic effect, was corrected with infusions of intravenous immune globulin. Patients were also treated for symptoms associated with tumour lysis syndrome, an effect of tumour breakdown. Novartis expects to initiate a Phase II clinical trial with CART-19 in collaboration with Penn during the fourth quarter of 2012.
"Initial data provide proof that this CAR therapy can activate a patient's own immune system to fight cancerous tumours," said Dr. Carl June, director of Translational Research and professor of Pathology and Laboratory Medicine in the University of Pennsylvania's Abramson Cancer Center and Perelman School of Medicine. "In partnering with Novartis, we aim to develop CAR therapies into commercial agents in the battle against cancer."
"Penn's intellectual resources combined with a pharmaceutical industry leader like Novartis offers a powerful symbiotic relationship in our mutual goal of finding more effective treatments for cancer," said Dr. J. Larry Jameson, 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 may open doors for use of this promising treatment option for 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 CARs developed through the collaboration for all indications and CART-19. In addition Novartis will provide an up-front payment, research funding, funding for the establishment of the CACT and milestone payments for the achievement of certain clinical, regulatory and commercial milestones and royalty payments.