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The first FDA-approved gene therapy for relapsed or refractory B-cell acute lymphoblastic leukemia (ALL)


Relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL)

What is KYMRIAH® (tisagenlecleucel)?

Immunotherapy is a form of treatment that uses the body’s immune system to treat cancer. There are several types of immunotherapy. KYMRIAH® (tisagenlecleucel) is a type of cellular immunotherapy that involves genetically modifying a patient’s T cells (a type of immune cell) with a specialized receptor engineered to target a marker on the surface of the Pre-B ALL cancer cells (in this case CD19). This specialized receptor is called a Chimeric Antigen Receptor, or CAR. Once the CAR is attached to the patient’s T cell, it is known as a CAR T cell. Because KYMRIAH® (tisagenlecleucel) involves genetic modification, it is considered a “gene therapy”. It is the first cellular immunotherapy treatment approved by the U.S. Food and Drug Administration (FDA).

There are many types of CAR T cells that target different cancers, but KYMRIAH® (tisagenlecleucel) only targets CD19, and is only FDA-approved for certain indications. FDA approval means a qualified doctor is able to prescribe this treatment. Because KYMRIAH® (tisagenlecleucel) is a specialized therapy, it is only available at certain centers, such as Texas Children’s Cancer Center.;

Who can receive KYMRIAH® (tisagenlecleucel)?

KYMRIAH® (tisagenlecleucel) is approved to treat patients under age 25 who have CD19-expressing B-cell acute lymphoblastic leukemia (ALL) that is:

  1. In second or greater relapse (has come back more than once)
  2. refractory (has not gone away despite other treatments)

KYMRIAH® is a potentially curative treatment option for patients whose cancer is no longer in remission; however, it can have severe or even life-threatening side effects. Read about some of the side effects of KYMRIAH® (tisagenlecleucel) on

What does KYMRIAH® (tisagenlecleucel) involve?

Step 1: The process of cellular immunotherapy including KYMRIAH® (tisagenlecleucel), begins with the patient. After ensuring a patient meets the prescribing criteria for KYMRIAH® (tisagenlecleucel), the patient will undergo a special procedure called leukapheresis. The purpose of leukapheresis is to collect the patient’s T cells in order to begin the manufacture process for KYMRIAH® (tisagenlecleucel). Prior to undergoing leukapheresis, patients first have a large IV or catheter placed (under anesthesia). Afterwards, they are connected to a pheresis machine (similar to a dialysis machine) in order to collect the T cells. The leukapheresis process takes around 4-6 hours and occurs in a special unit of the hospital. Once we confirm that we have collected enough T cells, the patient is disconnected from the pheresis machine and the catheter is removed. 

Step 2: The patient’s T cells are frozen in our laboratory, then sent to a special manufacturing facility where KYMRIAH® (tisagenlecleucel) manufacture occurs. The T cells are genetically modified with a special receptor designed to target CD19, present on the leukemia cells. This process can take several weeks, but patients/families are updated on the process along the way.

Step 3: While awaiting manufacture of KYMRIAH® (tisagenlecleucel), patients may require chemotherapy to help keep their leukemia under control.

Step 4: Treatment involves administration of KYMRIAH® (tisagenlecleucel), usually via an existing central line, after receiving several days of “conditioning” or “lymphodepleting” (space-making) chemotherapy. Patients may receive KYMRIAH® (tisagenlecleucel) in the outpatient clinic or in the hospital, and may require hospitalization to monitor for side effects afterwards.

Step 5: Patients are required to remain in the Houston area—no farther than a 1 hour commute to Texas Children’s Cancer Center—for a minimum of 4 weeks after receiving KYMRIAH® (tisagenlecleucel). Patients will have frequent follow-up visits in the clinic to monitor for side effects and response to therapy. Patients will usually undergo bone marrow and spinal fluid evaluation approximately 4 weeks after treatment with KYMRIAH® (tisagenlecleucel).

Detailed criteria are listed on and

KYMRIAH Referring Physician Guide
KYMRIAH Adolescent and Young Adult Guide


Rayne Rouce, MD

Bernadette Burttschell
Manager, Bone Marrow Transplant Operations

Miracle Okoye
Coordinator, Bone Marrow Transplant