Immunotherapy is a treatment that mobilizes the body’s own immune system to fight diseases. A new type of personalized immunotherapy — CAR T-cell therapy — is revolutionizing treatment for B-cell acute lymphoblastic leukemia (ALL), the most common pediatric cancer.

Most children with ALL can be cured using conventional treatments: chemotherapy, radiation and stem cell transplant. But in 20 percent of cases, the cancer is refractory (does not respond to treatment) or returns after treatment. Until now, doctors had few options to fight aggressive ALL. Today, the medical community sees great promise in CAR T-cell therapy for these patients.

CAR (Chimeric Antigen Receptor)T-cell therapy reprograms a patient’s disease-fighting white blood cells (T-cells) to seek out, recognize and attack cancer cells without harming healthy cells. After CAR T-cell immunotherapy saved the life of a young ALL patient in Philadelphia five years ago, the experimental treatment was offered to more children through clinical trials. In the majority of cases, signs of cancer disappeared and the patients continue to be in remission.

How Does CAR T-cell Therapy Work?

Clinical trials at UChicago Medicine helped develop CAR T-cell therapy for leukemia and lymphoma patients. By removing, supercharging, then returning white blood cells into the bloodstream with instructions to find and kill cancer cells, this living drug can often result in full remission. The process works like this, CAR T-cell therapy uses genetically engineered versions of the patient cells to find tumor cells and kill them with minimal damage to healthy cells.

In the first part of the process, T-cells, the workhorse of the immune system, are collected from the patient's blood. Then scientists insert instruction that enable those T-cells to find specific cancer cells. While the T-cells multiply in the lab, the patient receives chemotherapy to reduce the number of cancer cells. And finally, the engineer T-cells are returned to the patient's bloodstream, where they seek out and kill remaining cancer cells. For more information about this exciting new treatment, visit UChicagoMedicine.org/defeatcancer or call 844-482-7823

CAR T-cell therapy does present a risk of serious, potentially life-threatening side effects, which include high fever, flu-like symptoms, infections and low blood pressure. The FDA requires special certification for all sites offering the treatment to confirm the institution is well suited to handle serious adverse reactions, should they occur.

On August 30, 2017, the Food and Drug Administration approved CAR T-cell therapy as a treatment for advanced pediatric ALL. Comer Children’s is the first pediatric hospital in the Chicago region certified to offer CAR T-cell therapy.

"We are thrilled to offer CAR T-cell therapy to children in the Chicago area," said John M. Cunningham, MD, Comer Children’s Hospital physician-in-chief and director of the pediatric cellular therapy program. "It changes the face of treatment for pediatric ALL. And we believe CAR-T will be a foundation for other therapies that eradicate cancer."

CAR T-cell Therapy for Pediatric Patients

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