Cellular Therapy (Pediatric)

Cellular therapy is a form of therapy that uses a patient’s own living cells to target their cancer.

New forms of cellular therapy, such as CAR T-cell therapy, are quickly transforming the treatment of relapsed or resistant leukemia and lymphomas, using a donor’s own blood cells to target and fight cancer cells more effectively. University of Michigan Health was the first center in the state to use CAR T-cells to treat childhood leukemia, having begun our CAR-T therapy program in 2014. We were one of 15 sites in North America involved in the FDA licensing trial using CAR T-cells to treat B-cell leukemia in children. We are the largest pediatric CAR T-cell therapy program in the state and are proud to bring this novel therapy to children and young adults in our region.

What is Chimeric Antigen Receptor T-cell (CAR) T-cell Therapy?

Chimeric antigen receptor T-cell therapy, or CAR T-cell therapy, is a type of immune therapy that involves extracting billions of a patient’s own T-cells – disease-fighting white blood cells – which are then re-programmed through cellular engineering techniques to target specific cancer cells. The technique essentially transforms the patient’s cells into what scientists call "a living drug."

What are the Benefits of CAR T-cell Therapy?

Historically, treatment for leukemia has been heavily dependent on the use of chemotherapy. Unfortunately, the side effects of chemotherapy can be tremendous, as chemotherapy attacks cells both good and bad that are actively growing when the chemotherapy is given.

In contrast, CAR T-cell therapy trains a patient’s own white blood cells to target specific cancer cells, sparing normal, healthy tissue. Imagine an army of immune cells in your blood, trained to search and destroy your cancer cells. That is the idea behind CAR T-cell therapy.

Who can Benefit From CAR T-cell Therapy?

CAR T-cell therapy is a treatment option for children with B-cell acute lymphoblastic leukemia (ALL) that does not respond to treatment or has relapsed two or more times.  University of Michigan Health also offers CAR T-cell therapy for adults through the Rogel Cancer Center.

What is CAR T-cell Therapy Like for the Patient?

CAR T-cell therapy includes a number of steps.

  1. Evaluation: Patients referred to C.S. Mott Children’s Hospital for relapsed or resistant leukemia treatment will be evaluated to see if they are eligible for CAR T-cell therapy. This could involve a series of exams and tests, including blood tests, a bone marrow aspirate / biopsy, spinal tap (for leukemia patients), X-rays, and other scans.
  2. T-cell collection: If your child is determined to be a good candidate for CAR T-cell therapy, a portion of his or her T-cells will be removed through a blood draw process called apheresis.  During this outpatient procedure, a portion of your child’s T-cells (around 1%) are removed from the blood stream over a two to three-hour period. The process leaves the remainder of your immune system intact.
  3. Cell “training”: Once collected, the T-cells are then manufactured into the final CAR T-cell product through a two to four-week process in a lab. The T-cells are genetically modified – or “trained” – by producing a protein called chimeric antigen receptor (CAR) on the cell surface. The CAR receptor helps the white blood cells effectively target a protein on the leukemia cells and destroy only those cancerous cells.
  4. Reinfusion: The CAR T-cells are then given to patients through an IV infusion over approximately 5-10 minutes. Once the CAR-T cells are in the patient’s body, they begin to multiply and attack the cancer cells they have been programmed to destroy.
  5. Inpatient hospitalization: On average, patients will be admitted to the hospital for two to three weeks following the CAR T-cell infusion, so that the medical team can closely monitor for side effects during this period. One significant side effect of CAR T-cells is the potential development of cytokine release syndrome (CRS), sometimes called a cytokine storm.  The cytokine storm is caused by the immune system “revving up” to target the tumor cells. Symptoms can include fevers, shakes, chills, fluid retention, and changes in blood pressure.  In severe instances, it may require the patient be cared for in our pediatric intensive care unit (PICU). CRS typically develops within the first 1-2 weeks after the CAR T-cell infusion, with symptoms ranging from mild to severe, even life threatening in some patients. The medical team will monitor patients closely during this period and provide specialized treatment for severe forms of CRS if necessary.
  6. Discharge: After the patient is discharged from the hospital, they will need to stay within a 60-mile radius of the Mott for up to 8 weeks so that our clinicians can continue to monitor them closely and respond quickly to any side effects.

The Michigan Difference in cellular therapy

University of Michigan Health has been on the front lines of CAR T-cell therapy for the past several years, participating in the pivotal trials that led to the approval of the first FDA-approved CAR T-cell therapy in 2017.

Our cancer center is the first site in Michigan to offer both currently FDA-approved CAR T-cell therapies: Kymriah™ for leukemia and Yescarta™, a form of CAR T-cell therapy for adults with non-Hodgkin lymphoma.

University of Michigan C.S. Mott Children's Hospital is the largest, most experienced pediatric marrow transplant and cellular therapy program in the state of Michigan, with recognized expertise in both established transplant techniques and innovative research initiatives.

Our physicians specialize in the care of relapsed and resistant leukemia, offering access to the latest innovative treatment protocols and Phase 1 clinical trials.

Take the Next Step

To learn more about CAR T-cell therapy at C.S. Mott Children’s Hospital, call 734-936-9814.