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Latest results of investigational cellular therapy in pediatric acute lymphoblastic leukemia, and first results of lymphoma trial

The latest results of clinical trials of more than 125 patients testing an investigational personalized known as CTL019 was presented by a research team at the 56th American Society of Hematology Annual Meeting and Exposition. Highlights of the new trial results include a response rate of more than 90 percent among pediatric 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 team’s leader, 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 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 who were treated at the Children’s Hospital of Philadelphia (Abstract #380) includes data on 39 patients. The findings, which were 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 team’s report on 25 pediatric and five adult patients which was published recently in the New England Journal of Medicine.

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.