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Rady Children's Specialists

KYMRIAH: CAR T-Cell Therapy

Diagnosed at age 12 with ALL, Abegail was treated but relapsed twice. Thanks to CAR T-cell therapy, she is currently cancer-free and feeling happy and healthy.

Rady Children’s Hospital is a certified CAR T-cell treatment center. This enables us to provide the Food and Drug Administration-approved CAR T-cell therapy called KYMRIAH (tisagenlecleucel) to cancer patients who are up to 25 years old with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.

CAR T-cell therapy is part of our Blood and Bone Marrow Transplantation Program. Deborah Schiff, M.D., a member of this BMT team, oversees the treatment. Read the news release.

About ALL

ALL is the most common childhood leukemia, with approximately 3,100 patients age 20 and younger being diagnosed each year, according to the National Cancer Institute. Approximately 85 percent of ALL in children is B-cell. Cure rates and survival outcomes for patients with ALL have improved dramatically over the past several decades, particularly among children. But in about 15-20 percent of patients, the cancer does not respond to standard chemotherapy or returns after initial treatment.

What is CAR T-cell therapy?

CAR (chimeric antigen receptor) T-cell therapy is a promising gene therapy that uses modified versions of a patient’s own blood cells to target and destroy cancer cells. It involves three types of innovative technologies:

  1. Cellular therapy – using the patient’s own cells as therapy
  2. Gene therapy – inserting genes into a patient’s cells, thereby causing these cells to produce a new therapeutic protein
  3. Immunotherapy – harnessing the patient’s own immune system to treat his/her disease

How does it work?

A process called leukapheresis is used to collect the patient’s own T-cells intravenously. These cells are genetically engineered to target a protein called CD19. The new therapeutic cells are then reinfused to the patient via an intravenous line. Once returned to patient’s body, the CAR T-cells target and kill the leukemia cells that express CD19.

What should you expect?

Your doctor will discuss potential reactions and side effects.

Financial assistance and patient support

Through the KYMRIAH CARES program, Novartis offers information on financial assistance as well as patient support programs to help patients and families access this therapy. Services may include transportation, lodging and healthy meals. Learn more here.

For more information about whether KYMRIAH is right for your child or to refer a patient, contact Dr. Schiff at

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