Oncologists from Children’s Hospital of Philadelphia (CHOP) today celebrated a watershed moment in medicine: approval by the European Commission (EC) of Kymriah (tisagenlecleucel, formerly CTL019) — the first-ever U.S. Food and Drug Administration (FDA) approved personalized CAR T-cell gene immunotherapy for aggressive blood cancers, pioneered together with Novartis and the Perelman School of Medicine at the University of Pennsylvania.
The EC approved Kymriah for the treatment of pediatric and young adult patients up to 25 years of age with B-cell acute lymphoblastic leukemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse; and for the treatment of adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy.
“Six years ago, when we treated the first child, Emily Whitehead, with this experimental therapy, these patient populations had exhausted all the treatments we had to offer. There was nothing left for us to offer,” said Stephan Grupp, MD, PhD, Director of the Cancer Immunotherapy Program and Section Chief of Cell Therapy and Transplant at CHOP, and a Professor of Pediatrics in the Perelman School of Medicine at the University of Pennsylvania. “In a remarkably short period of time we’ve ushered in a brand new field of medicine. We couldn’t be more pleased the CAR T-cell treatment we first gave Emily has not only saved her life, but has been proven effective in more blood cancers, and may completely change the outcomes of these diseases in patients in Europe and around the world.”
Chimeric antigen receptor T (CAR-T) cell therapy genetically modifies a patient’s immune cells to make them seek out and kill leukemia cells. The approach was developed by a team led by Carl June, MD, of the Perelman School of Medicine at the University of Pennsylvania. In 2012, Penn and Novartis entered into a global collaboration to further research, develop and commercialize Kymriah. CHOP was the first institution to use the therapy in children with leukemia.
“To date, our unrivaled immunotherapy program has now treated more than 250 patients with this innovative therapy, more than any other pediatric institution in the world,” said Stephen Hunger, MD, Chief of the Division of Oncology and Director of the Center for Childhood Cancer Research at CHOP. “Young cancer patients are in critical need of personalized cell therapies here in the U.S., and we’re delighted to now see Kymriah fill an unmet need abroad.”
EC approval of Kymriah was based on two pivotal Novartis-sponsored global, multi-center Phase II trials, ELIANA and JULIET, conducted in countries worldwide, including the U.S. and Europe.
“ELIANA data evaluated by the European Medicines Agency demonstrate strong and durable response rates,” said ELIANA lead study author Shannon L. Maude, MD, PhD, a pediatric oncologist at CHOP and Assistant Professor of Pediatrics in the Perelman School of Medicine at the University of Pennsylvania. “Patients not only get better, but personalized, cancer-fighting cells can remain in the body for months or even years, effectively doing their job.”
The U.S. FDA approved Kymriah to treat children and young adults with r/r ALL in August 2017, and then, in May 2018, approved it for the treatment of adults with r/r large B-cell lymphomas. CHOP offers Kymriah for both r/r ALL and r/r DLBCL.
Clinical research trials are also underway at CHOP to further improve CAR T-cell therapy and apply it for use in more pediatric cancers. Find more information on our Cancer Immunotherapy Program.
Editor’s Notes: Some CHOP investigators serve or have served as consultants to Novartis to support the company’s continued development of this treatment. In addition, Dr. Grupp is an inventor of a process for treating toxic reactions to the therapy.
The University of Pennsylvania has licensed some technologies involved in these studies to Novartis. Some of the scientists involved in these trials are inventors of these technologies. As a result of the licensing relationship with Novartis, the University of Pennsylvania receives significant financial benefit, and some of these inventors have benefitted financially and/or may benefit financially in the future.
Contact: Amy Burkholder, The Children’s Hospital of Philadelphia,