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EMBARGOED FOR RELEASE UNTIL: Abstract #380: 10 a.m. PST, Saturday, Dec. 6 Abstract #1982 and #1983: 5:30 p.m. PST, Saturday, Dec. 6 Abstracts #3087 and #2296: 6:00 p.m. PST, Sunday, Dec. 7

Newswise SAN FRANCISCO The latest results of clinical trials of more than 125 patients testing an investigational personalized cellular therapy known as CTL019 will be presented by a University of Pennsylvania research team at the 56th American Society of Hematology Annual Meeting and Exposition. Highlights of the new trial results will include a response rate of more than 90 percent among pediatric acute lymphoblastic leukemia patients, and results from the first lymphoma trials testing the approach, including a 100 percent response rate among follicular lymphoma patients and 45 percent response rate among those with diffuse large B-cell lymphoma.

We have now treated more than 125 patients in our trials of the chimeric antigen receptor (CAR) therapy CTL019, and with each patient, we learn more and more about the potential of this therapy, said the research teams leader, Carl June, MD, the Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine in Penns Perelman School of Medicine, and director of Translational Research in the Abramson Cancer Center. We are continuing to refine our approach to ensure the best outcomes for patients who may be eligible for this experimental therapy, and we hope our findings will contribute to the emerging field of cellular therapy as a whole.

This personalized cellular therapy approach begins with patients own immune cells, collected through a procedure similar to dialysis. 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.

Updated results of a CTL019 trial for children and young adults with relapsed, treatment-resistant acute lymphocytic leukemia who were treated at the Childrens Hospital of Philadelphia (Abstract #380) includes data on 39 patients. The findings, which will be presented by Stephan Grupp, MD, PhD, the Yetta Deitch Novotny Professor of Pediatrics and director of Translational Research in the Center for Childhood Cancer Research at the Children's Hospital of Philadelphia, build on the teams report on 25 pediatric and five adult patients which was published in the New England Journal of Medicine in October.

Thirty six of 39 children (92 percent) achieved a complete response (CR) after receiving an infusion of the modified cells. After a median follow-up of six months, more than two-thirds (70 percent) of children who responded remained in remission and 75 percent were alive, including the first patient to receive the therapy, in the spring of 2012. These results were achieved with only 3 of the patients going on to receive stem cell transplant while in remission.

All pediatric patients who responded to the therapy experienced a cytokine release syndrome (CRS) within a few days after receiving their infusions a key indicator that the engineered cells have begun proliferating and killing tumor cells in the body, but also a known potentially lethal type of toxicity. Patients who experience a CRS typically have varying degrees of flu-like symptoms, with high fevers, nausea, muscle pain, and sometimes, low blood pressure and breathing difficulties. Some patients require treatment with anti-cytokine agents and steroids to manage these symptoms.

The research team will also report the first results of a CTL019 study of patients with relapsed or refractory non-Hodgkin lymphomas (NHL) (Abstract #3087). In patients with follicular lymphoma (FL) or diffuse large B cell lymphoma (DLBCL) who received infusions of CTL019, assessments at three months after treatment revealed that all five FL patients (100 percent) and five out of 11 DLBCL patients (45 percent) responded to the therapy, including complete responses in four patients (80 percent) with FL and four patients (36 percent) with DLBCL. All patients who received infusions developed varying degrees of CRS. The longest complete response durations are ongoing, at 8.8 months for DLBCL and 7.4 months for FL; all other responses continue, as well. The findings will be presented by Jakub Svoboda, MD, an assistant professor of Medicine in the Abramson Cancer Center, on behalf of the Lymphoma Program under the leadership of the studys principal investigator, Stephen J. Schuster, MD, the Robert and Margarita Louis-Dreyfus Associate Professor of Chronic Lymphocytic Leukemia and Lymphoma.

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Penn Medicine Researchers Announce Latest Results of Investigational Cellular Therapy CTL019