University Of Pennsylvania’s T-Cell Therapy Shows Promising Results

By C. Rajan, contributing writer

The University of Pennsylvania has announced promising results of its novel chimeric antigen receptor (CAR) therapy for cancer.

In the study involving 25 children and five adults with end-stage acute lymphoblastic leukemia (ALL), there was an impressive 90 percent response rate with complete remission.

Twenty-seven of the 30 patients went into complete remission after receiving the investigational therapy (called CTL019), and 78 percent of the patients were alive six months after treatment. The longest remission among the patients has lasted almost three years.

The patients who participated in these trials had relapsed as many as four times, including 60 percent whose cancers came back even after stem cell transplants. Their cancers were so aggressive they had no treatment options left, said the studys senior author, Stephan Grupp, MD, PhD, at the Children's Hospital of Philadelphia. The durable responses we have observed with CTL019 therapy are unprecedented.

The ongoing study is being conducted by researchers at the Childrens Hospital of Philadelphia and the Hospital of the University of Pennsylvania (Penn). The CAR trial program enrolling children with leukemia is also expanding to nine other pediatric centers.

The experimental CAR therapy received FDAs breakthrough designation in July for the treatment of relapsed and refractory adult and pediatric ALL. The novel treatment was pioneered by Penn researchers and then supported by Novartis. Penn entered an exclusive global research and licensing agreement with Novartis in 2012 to develop and commercialize personalized CAR T-cell therapies for cancers.

"This represents a really powerful therapy for ALL," Penn oncologist David Porter says. "We've treated enough patients to confirm that. It's time to start multi-center trials."

A CAR is a genetically engineered marker protein that is grafted onto T cells, which are part of the immune system. The CAR activates the T cell to attack tumor cells that express specific markers; in this case, the target is a protein called CD19.

The treatment procedure involves removing patients' T cells via an apheresis process and then genetically reprogramming them to hunt tumor cells. When injected back into patients bodies, these new hunter cells multiply and attack tumor cells expressing CD19. The hunter cells can grow, creating 10,000+ new cells in the body for each single engineered cell injected into the patients.

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University Of Pennsylvania's T-Cell Therapy Shows Promising Results