Advancing Innovative Cancer Therapies

Advancing Innovative Cancer Therapies
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Novartis and University of Pennsylvania 

In August 2012 the Novartis Institutes for Bio-Medical Research, based in Cambridge, Massachusetts,
and the University of Pennsylvania’s Perelman School of Medicine (Penn Medicine) announced they had entered into an exclusive global research and licensing agreement. Under the alliance, Penn Medicine granted Novartis an
exclusive worldwide license to the technologies used in an ongoing trial of patients with chronic lymphocytic leukemia (CLL) as well as future therapies based on chimeric antigen receptor (CAR) technology developed through the collaboration. As part of the agreement, Novartis invested $20 million to establish the $27-million Center for Advanced Cellular Therapies (CACT) and to support future research in CAR technology. Construction on the CACT—formally known as the Novartis-Penn Center for Advanced Cellular Therapeutics—began in December 2014, and
the facility opened in early 2016. It adjoins the existing cancer therapeutics floor in the Smilow Center for Translational Research, allowing it to be fully integrated into Penn Medicine’s research and clinical operations. The CACT employs 100 highly specialized professionals working across 6,300 sq. ft. of cleanroom space specially designed for cell engineering and 24,000 sq. ft. of laboratory and cell therapy manufacturing space
with the capacity to manufacture cellular therapies for up to 400 patients per year. “The CACT will allow us to leverage this progress to develop and test new approaches more quickly,” says Carl H. June, MD, the Richard W.
Vague Professor in Immunotherapy at Penn Medicine. “At the same time, we’ll be able to expand our ability to manufacture personalized cell therapies for a greater number of trials.”


Although the CACT is primarily a lab and research space, the project design team brought in experts from outside the fields of science and technology with a view to making the facility more efficient and effective. They also included specialists in corporate workplace design to create the best environment possible for employees. Open-plan laboratories within the facility allow researchers from the center’s four principal investigator (PI) teams to work together rather than in silos, as in many lab facilities. Each PI team works on di¤erent aspects of the research, which
requires specialized facilities: the clinical cell and vaccine production facility, a quality control lab, a transformational and correlative studies lab, and a product development lab. This approach promotes what the center calls a “new scientific workplace culture,” which blurs the boundaries across disciplines and encourages collaboration. The center was designed and built to meet current good manufacturing practice cleanroom
standards, including ISO Class 10,000 cell processing rooms and ISO Class 100,000 support spaces. The flow of specimens, materials, and staff has also been carefully considered to ensure the highest standard of cleanliness
throughout the facility. These and other design decisions are intended to improve collaboration and communication
among the teams, helping them increase speed to market and produce individual patient therapies more efficiently. The strategic layout and organization of lab equipment will also dramatically cut the time it takes to generate “hunter” cells: While it previously took a month to produce these therapeutic agents for each patient, the new facility will help reduce the time to two weeks. The CACT advances this process through the different functional areas contained within the facility, such as vaccine development, assay development, and correlative studies of blood and other bio specimens, to examine how trial participants respond to the therapies they receive.


In addition to continued trials in CLL, Penn has engineered T cell trials underway for other types of leukemia, as well as lymphoma, mesothelioma, myeloma, and neuroblastoma. In 2014, the US Food and Drug Administration awarded breakthrough therapy designation to CTL019, an investigational CAR therapy for the treatment of relapsed and refractory adult and pediatric acute lymphoblastic leukemia. CTL019 is the first personalized cellular therapy for the treatment of cancer to receive this important classification. Pioneered by Penn Medicine, this new application first removes a patient’s T cells, then genetically reprograms them in the CACT’s clinical cell and vaccine production facility. After being infused back into the patient, these hunter cells both multiply and attack, targeting tumor cells that express the DCD19 protein. Tests reveal that each single engineered cell can grow into an army of more than 10,000. Ultimately, the CACT’s objective is to develop new treatment options for life-threatening diseases. “This new joint center is testimony to the power that comes from merging academic discovery and the generation of new
medicines,” says Mark Fishman, President of Novartis Institutes for BioMedical Research.