In an effort to get FDA approval for a new cancer therapy, pharmaceutical giant Novartis is teaming with the University of Pennsylvania and investing at least $20 million in a new center to expand the university's work.
The collaboration between Novartis and Penn is expected to start in the fall with a new research and development facility, the Center for Advanced Cellular Therapies, to be established within the next year. The center's location has not yet been determined but will most likely be on Penn's campus, university officials said Sunday.
The partnership comes after a Penn team led by immunologist and gene-therapy pioneer Carl June published work last year in two major journals showing that it had genetically engineered patients' T cells - the big guns of the immune system - to recognize and attack the malignant cells of chronic lymphocytic leukemia, and then stand guard against the disease.
Although only three patients who had failed standard therapies were treated with the designer T cells, all three went into lengthy remission. Never before in published experiments had engineered T cells multiplied - and then persisted - enough to be so effective in patients.
David Strayer, a professor of pathology at Thomas Jefferson University who has long studied gene therapy, said the Penn and Novartis agreement comes "at a time when research funding is quite difficult to garner."
The partnership is an "encouraging sign" that large pharmaceutical companies are willing to team with medical schools.
"This could be the tip of the iceberg in pharmaceutical companies' having an interest in products being designed and developed by medical schools," Strayer said.
For all their sophistication, the designer T cells - with what scientists call chimeric antigen receptors (CAR) - remain experimental. The new T cells kill both healthy and cancerous B cells, so patients' supply can be permanently depleted.
B cells, which help fight viral and bacterial infections, are not indispensable to the body. But patients who lack B cells need regular intravenous doses of immunoglobulins to reduce their chances of infection.
In an editorial in the New England Journal of Medicine, two oncologists, who called the results of the new study impressive, also warned that toxic effects, known and unknown, "could pose substantial problems."
Finding the right dose of T cells and continuing to work on the impacts of dying cancer cells - known as tumor lysis syndrome - are June's two main goals for the launch of the research partnership. "A lot of new science will come out of this, which is pretty exciting," he said.
After June's team findings were published last year, four or five pharmaceutical companies contacted Penn to ask about the possibility of teaming on future immunotherapy work.
Penn ultimately chose Novartis because of its oncology background, June said.
Under terms of the agreement, Novartis will get an exclusive worldwide license to the technologies used in the treatment of patients with chronic lymphocytic leukemia (CLL) as well as future CAR-based therapies developed through the collaboration. Additional milestone and royalty payments to Penn are also part of the agreement, though neither side would disclose them.
"Immunotherapy is one of the exciting frontiers in cancer research, and the CAR technology developed by Penn has shown early promise as a new way for treating cancer," Novartis spokeswoman Rachel Spielman said.
So far, June and his team have treated 10 leukemia patients, including one child. All have had positive results, June said. The first item on the agenda for the new partners is to kick off a pilot clinical trial for the CAR therapies to get FDA approval of the modified T cell therapy.
Though the initial focus of the center will be leukemia patients, both adults and children, June said he hoped to start working on using CAR therapy for ovarian- and brain-cancer patients.
The group of scientists dedicated to Penn's research and use of CAR technology is expected to increase from 12 to about 80, June said. Once more staff is hired for the center, at least 50 patients will be treated in a year.
The ideal candidate right now has progressive leukemia, which has basically been deemed incurable, June said.
"We are hoping we can start earlier," when the cancer has not progressed as much, June said. He also said his team would begin to treat other cancers in small pilots this year.
The long-term goal is to commercialize novel CAR-based immunotherapies so patients at community hospitals all over the country can have access to Penn's breakthrough.
"There's an ethical imperative to get it out to other people," June said.
University officials say the venture will bring full circle the 1960 discovery of the Philadelphia chromosome, the first description of a chromosome abnormality that causes cancer.
"For us scientists and doctors, we want to improve treatments available," said J. Larry Jameson, dean of the Perelman School of Medicine at Penn and executive vice president of the health system. "This kind of therapy has broader application to the field of cancer."