Norton Children’s Cancer Institute, affiliated with the UofL School of Medicine, offers chimeric antigen receptor (CAR) T-cell therapy for children and teens with fairly advanced and/or recurrent B-cell acute lymphoblastic leukemia (ALL). CAR T-cell therapy is a newer approach to cancer treatment that uses the body’s own immune cells to kill cancerous cells. Norton Children’s Cancer Institute is the only pediatric program in Kentucky offering CAR T-cell therapy.

CAR T-cell therapy was approved by the U.S. Food and Drug Administration (FDA) for treating relapsed B-cell ALL in 2017. At this time, CAR T-cell therapy is not used for other types of leukemia or childhood cancer, but research is ongoing to expand this therapy to other types of childhood cancer.

What Is CAR T-cell Therapy?

CAR T-cell therapy is an immunotherapy in which the body’s immune system is activated to fight off disease. CAR T-cell therapy teaches the body’s immune system to recognize and eliminate cancerous B-cells, which can help the body control the growth of cancer cells over time.

How CAR T-cell Therapy Works

CAR T-cell therapy works as follows:

• T-cells are taken from an ALL patient’s blood through apheresis. T-cells are a type of white blood cell that works to eliminate disease from the body.
• The T-cells are genetically modified in a lab and “taught” to express a protein that recognizes and binds to CD19, a marker found on cancerous B-cells.
• The T-cells taught to recognize cancerous B-cells, called “CAR T-cell product,” are injected back into the patient’s body.
• The modified T-cells begin to attach to the cancerous B-cells, destroying the cancer cells.
• The modified T-cells also begin multiplying in the body, which can help the body continue to fight cancerous B-cells long after therapy ends.

Children and teens who receive CAR T-cell therapy will continue to be monitored for side effects after therapy.

We Offer FDA-approved Therapy

Norton Children’s Cancer Institute offers tisagenlecleucel (trade name Kymriah), an FDA-approved CAR T-cell treatment. This course of treatment is offered to patients up to age 25 with B-cell precursor ALL refractory (meaning treatment is not responding) or in second or later relapse.

We are the first and only select treatment center offering Kymriah to pediatric patients in Kentucky.

Ask Your Physician About CAR T-cell Therapy

Families are encouraged to ask about potential benefits of cellular therapy early on during a child’s cancer treatment process. For CAR T-cell therapy, collecting cells from a patient before serious illness and/or intensive chemotherapy can help the therapy be as successful as possible. About 85% of ALL cases are successfully treated with standard chemotherapy. However, in the other 15% of cases, chemotherapy works temporarily or not at all. CAR T-cell therapy is not a treatment for newly diagnosed leukemia — only for patients whose ALL is not responding to chemotherapy and the disease may have returned after a bone marrow transplant.