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:
- In second or greater relapse (has come back more than once)
- 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).
Rayne Rouce, MD
Manager, Bone Marrow Transplant Operations
Coordinator, Bone Marrow Transplant