CAR-T Therapy for Children with Relapsed or Refractory Acute Lymphoblastic Leukemia (ALL)
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A promising new treatment, known as chimeric antigen receptor or CAR-T cell therapy, is now available at American Family Children’s Hospital for children with acute lymphoblastic leukemia (ALL) that either did not respond to standard therapy or has relapsed a second time.
This groundbreaking cancer treatment, called Kymriah (tisagenlecleucel), is a form of immunotherapy, that received approval from the U.S. Food and Drug Administration (FDA) in August 2017 after 83 percent of children were found to be in a complete remission within 3 months after receiving CAR-T. This remission rate was more than double any prior FDA-approved medication for this disease.
CAR-T cell therapy “supercharges” the child’s own infection-fighting white blood cells (called T-cells) to seek out and destroy leukemia cells that display a specific protein marker on their cell surface. The child’s own T cells are removed from the blood and sent to a processing center for treatment and then shipped back to American Family Children's Hospital, where the child receives the “supercharged” T cells through infusion.
The collection of the child’s T-cells and the infusion of the CAR-T cells are both outpatient procedures. The infusion itself only takes a few minutes, but children are watched carefully for the first month and admitted to the hospital for any signs of serious side effects.
American Family Children’s Hospital is a research pioneer in childhood cancer and welcomes referrals of new patients who may qualify for CAR-T cell therapy.
“The early results of CAR-T cell therapy are very encouraging,” says Christian Capitini, MD, site principal investigator of UW-Madison’s pediatric cancer team, which is one of 13 U.S. sites that tested CAR-T cell therapy in clinical trials.
“Now that CAR-T cell therapy is FDA approved, it will become standard of care for children with refractory leukemia or with their second relapse, even after failing a bone marrow transplant,” Capitini said. “Because of the capacity of T cells to form memory, some of these children can be living years after infusion, without any more cancer treatment.”
CAR-T cell therapy may have unique side effects that are explained before treatment. They range from high fevers and body aches, to complications such as maintaining safe blood pressures and need for a ventilator. The side effects are treated with drugs, and the drug Actemra (tocilizumab) received expanded approval to help treat the side effects. Afterward, follow-up care involves an outpatient monthly infusion to prevent infections.
Dr. Christian Capitini Explains CAR-T Cell Therapy Treatment for Children
Why Your Child Should Come to UW Health's American Family Children's Hospital in Madison, Wisconsin