With an overall remission rate of 83%, the FDA approves CAR-T cell therapy for children and young adults with B-Cell ALL leukemia.

Swiss-based Novartis ushered in a new era in oncological therapeutics with the announcement, the FDA approved its chimeric antigen receptor T cell (CAR-T) therapy KymriahTM (tisagenlecleucel) for the treatment of children and young adults with B-cell precursor acute lymphoblastic leukemia (ALL) that is either refractory or in subsequent relapse. Novartis’ approval marks the launch of this class of one-time gene-transfer treatments that employs a patient’s T-cells to target and kill cancers, particularly in the blood.

To support the safe delivery of the therapy, Novartis said it is establishing a network of certified cancer centers throughout the U.S. to be staffed with professionals fully trained to administer the treatment. Joseph Jimenez, CEO of Novartis, explained the impact the approval has had on the company’s legacy. "At Novartis, we have a long history of being at the forefront of transformative cancer treatment. Five years ago, we began collaborating with the University of Pennsylvania and invested in further developing and bringing what we believed would be a paradigm-changing immunocellular therapy to cancer patients in dire need. With the approval of Kymriah, we are once again delivering on our commitment to change the course of cancer care."

Novartis’ treatment is first to market and use the 4-1BB costimulatory domain in its chimeric antigen receptor to enhance cellular expansion and persistence, said the company. "Tisagenlecleucel is the first CAR-T therapy to demonstrate early, deep and durable remission in children and young adults with relapsed or refractory B-cell ALL," said Stephan Grupp, Professor of Pediatrics at Penn and Director of the Cancer Immunotherapy Frontier Program at Children's Hospital of Philadelphia (CHOP). "We've never seen anything like this before and I believe this therapy may become the new standard of care for this patient population."