07:09am Friday 20 October 2017

Penn Medicine, Abramson Cancer Center Team Continues Progress in Investigational Gene Therapy for Blood Cancers

CTL019, a chimeric antigen receptor (CAR) therapy, begins with patients’ own immune cells, collected through a procedure called apheresis. The cells are then engineered in a laboratory and infused back into patients’ bodies after being trained to hunt and kill their cancer cells. All patients who enroll in the trials have cancers that have progressed despite multiple conventional therapies.

“We are proud to have successfully treated patients with advanced leukemia and lymphoma and to continue refining this therapy and developing new CARs to treat other types of cancers,” said Carl June, MD, the Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine in Penn’s Perelman School of Medicine, and director of Translational Research in the Abramson Cancer Center. “We were pleased in July 2014 when the U.S. Food and Drug Administration designated CTL019 as a ‘breakthrough therapy’ for relapsed and treatment-resistant acute lymphoblastic leukemia (ALL) in children and adults and are moving forward with our efforts to help even more patients.”

The team’s new patents for CTL019 include:

  • Method of treating leukemia using an anti-CD19 CAR. US Patent No. 8,906,682, issued December 9, 2014,  covers a method of treating leukemia comprising administering to a patient a pharmaceutical composition comprising an anti-tumor effective amount of a population of human T cells, wherein the T cells comprise a nucleic acid that encodes an anti-CD19 chimeric antigen receptor (CAR) having certain characteristics.
  • Composition covering a human T-cell expressing an anti-CD19 CAR. US Patent No. 8,911,993, issued December 16, 2014,  covers a pharmaceutical composition comprising an anti-tumor effective amount of a population of human T cells, wherein the T cells comprise a nucleic acid sequence that encodes an anti-CD19 chimeric antigen receptor (CAR) having certain characteristics.

Additionally, the team has received a patent for related anti-CD19 CAR research:

Method of treating leukemia using an anti-CD19 CAR containing a CD27 costimulatory signaling region. US Patent No. 8,916,381, issued December 23, 2014, covers a method of treating leukemia comprising administering to a patient a pharmaceutical composition comprising an anti-tumor effective amount of a population of modified autologous human T cells, wherein the T cells comprise a nucleic acid that encodes an anti-CD19 chimeric antigen receptor (CAR) having certain characteristics including a CD27 costimulatory signaling region.

Co-inventors of the new technologies include Carl June, MD, Bruce Levine, PhD, the Barbara and Edward Netter Professor in Cancer Gene Therapy in the department of Pathology and Laboratory Medicine, David Porter, MD, the Jodi Fisher Horowitz Professor in Leukemia Care Excellence and director of Blood and Marrow Transplantation in the Abramson Cancer Center, and Michael Milone, MD, PhD, an assistant professor of Pathology and Laboratory Medicine.

In August 2012, Penn announced an exclusive global research and licensing agreement with Novartis to further study, develop and commercialize personalized chimeric antigen receptor (CAR) T cell therapies for the treatment of cancers. 

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Penn Medicine is one of the world’s leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of the Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania (founded in 1765 as the nation’s first medical school) and the University of Pennsylvania Health System, which together form a $4.9 billion enterprise.

The Perelman School of Medicine has been ranked among the top five medical schools in the United States for the past 17 years, according to U.S. News & World Report‘s survey of research-oriented medical schools. The School is consistently among the nation’s top recipients of funding from the National Institutes of Health, with $409 million awarded in the 2014 fiscal year.

The University of Pennsylvania Health System’s patient care facilities include: The Hospital of the University of Pennsylvania — recognized as one of the nation’s top “Honor Roll” hospitals by U.S. News & World Report; Penn Presbyterian Medical Center; Chester County Hospital; Penn Wissahickon Hospice; and Pennsylvania Hospital — the nation’s first hospital, founded in 1751. Additional affiliated inpatient care facilities and services throughout the Philadelphia region include Chestnut Hill Hospital and Good Shepherd Penn Partners, a partnership between Good Shepherd Rehabilitation Network and Penn Medicine.

Penn Medicine is committed to improving lives and health through a variety of community-based programs and activities. In fiscal year 2014, Penn Medicine provided $771 million to benefit our community.


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