CAR-T therapy in pediatric leukemia

We stand at the crossroads of medical innovation, where cutting-edge scientific discoveries intersect with the resilience of the human body, providing hope to families grappling with a diagnosis of pediatric leukemia.

In an editorial paper, published in Oncoscience and titled “The price of hope: CAR-T therapy in pediatric leukemia,” researcher Alex Hoover from the University of Minnesota Medical School’s Division of Pediatric Blood and Marrow Transplantation discusses the chimeric antigen receptor T-cell (CAR-T) therapy tisagenlecleucel (tisa-cel)—a groundbreaking development in the treatment of B-cell lineage acute lymphoblastic leukemia (B-ALL) (the most common childhood cancer).

Following the pivotal ELIANA trial, tisa-cel was approved in the United States for the treatment of refractory or second or greater relapse of B-ALL in patients under age 25.

This innovative therapy involves genetically modifying a patient’s native T-cells—immune cells with the ability to kill and/or regulate infected or diseased cells—to express a receptor that targets cancer cells. The tisa-cel product is specifically engineered to target CD19 on B-lineage cells and has shown promising results in a historically challenging relapsed and refractory population.

Upon its approval in 2017, Novartis priced tisa-cel at $475,000—a number that many health care systems and payers balked at initially—and in 2023 the price increased to $508,250. However, compared to more recently approved ex vivo gene therapies such as bluebird bio’s Zynteglo for beta thalassemia priced at $2.8 million or Vertex Pharmaceuticals’ Casgevy for sickle cell disease at $2.2 million, the half-million dollar tisa-cel price tag is less staggering.

Importantly, the listed cost of these therapies includes only the product and does not include the health care cost for other necessary care including leukapheresis, chemotherapy administration and adverse event monitoring and management.

“Our team recently published an in-depth analysis of the comprehensive cost and utilization of commercial CAR-T therapy in pediatric B-ALL patients with commercial insurance in the United States,” say Hoover.

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