Cellular Immunotherapy Program

Cook Children's Cellular Immunotherapy Program provides individualized treatment that specifically targets a patient's cancer, with the goal of decreasing side effects and improving cure rates. Our program is unique because patients are managed by a multidisciplinary team including doctors who specialize in cellular immunotherapy for leukemia and bone marrow and stem cell transplant, along with dedicated nurses, child life specialists, social workers and financial counselors.

Cook Children's Hematology and Oncology Center is committed to providing leading edge treatments for pediatric cancer. Our team believes that the future of cancer treatment is through the use of targeted therapy such as immunotherapy whereby a patient's immune system is utilized to eradicate their cancer. We're excited to be able to offer a form of cellular therapy where patients are treated with cells that have been genetically reprogrammed with a receptor that targets their cancer cells, known as chimeric antigen receptor T-cell (CAR T-cell) therapy.

Why CAR T-cell therapy?

CAR T-cell therapy is especially promising for children and young adults with either relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL). B-ALL is the most common childhood leukemia, with B-cell making up approximately 85% of cases. And while cure rates have improved over the years, some 10-15% of patients don't respond to traditional treatments or relapse. CAR T-cell therapy offers these patients improved outcomes. Eligible patients at Cook Children's are able to receive KYMRIAH^® which is the first FDA-approved CAR T-cell therapy and the only FDA approved CAR T-cell for pediatric cancer patients.

What is KYMRIAH?

KYMRIAH is a CAR T-cell that is manufactured using T-cells collected from the patient. Then the cells are reprogrammed to target CD-19 which is a marker on the surface of B-ALL cells.

Who can receive KYMRIAH?

Children and young adults (up to age 25) with relapsed and/or refractory B-ALL may be eligible for treatment with KYMRIAH. In addition, B-ALL patients enrolled on the St. Jude Total XVII trial who meet specific eligibility criteria may be eligible to receive KYMRIAH as part of the Total XVII trial.

What is the objective of treatment with KYMRIAH?

The goal of treatment with KYMRIAH is to achieve remission.

How does treatment with KYMRIAH work?

Potential patients will have a consultation appointment with our Cellular Immunotherapy Program team where the patient's medical history will be reviewed to determine if they meet criteria to be treated with KYMRIAH. Then the individual will undergo apheresis which is a 3 to 6 hour process where a machine is used to collect T-cells from the patient's blood. After the T-cells are collected they will be sent to a lab where the cells are reprogrammed into CAR T-cells. This process takes approximately one month. Once the CAR T-cells have been manufactured the patient will receive a short course of chemotherapy to prepare the body to receive the cells. Then the cells are administered back to the patient. The patient is then closely monitored by our cellular immunotherapy team and repeat testing is performed to determine whether the patient is in remission.

What are the expected/long-term outcomes?

In the clinical trial leading to the FDA approval of KYMRIAH 81% of B-ALL patients treated with KYMRIAH achieved remission. (Maude SL, Laetsch TW, Buechner J, et al. Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia. N Engl J Med 2018;378:439-48.) Some patients treated with KYMRIAH may not require any additional treatment.

Questions about this program?

If you have questions about CAR T-Cell Therapy or Cellular Immunotherapy, please contact Holly Pacenta, M.D. You can also learn more about Dr. Pacenta here.

We are here to help.

If your child has been diagnosed, you probably have lots of questions. We can help. If you would like to schedule an appointment, refer a patient or speak to our staff, please call our offices at 682-885-6152.