KYMRIAH

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

Diseases

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)
   OR 
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 us.kymriah.com.

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 fda.gov and us.kymriah.com

A Caregiver's Guide to KYMRIAH Therapy (English | Spanish)
KYMRIAH Referring Physician Guide
KYMRIAH Adolescent and Young Adult Guide