Cellular Therapy (Pediatric)

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

Bone marrow transplantation is one type of cellular therapy that has become a mainstay of cancer treatment.

New forms of cellular therapy, such as CAR-T therapy, are quickly transforming the treatment of relapsed or resistant leukemia, helping a donor’s own blood cells target and fight cancer cells more effectively.  Michigan Medicine was the first center in the state to use CAR-T cells to treat childhood leukemia, having first administered CAR-T cells to a pediatric patient in 2014.

What is chimeric antigen receptor T-cell (CAR-T) therapy?

Chimeric antigen receptor T-cell therapy, or CAR T-cell therapy, is a type of immunotherapy that involves extracting billions of a patient’s own T-cells – disease-fighting white blood cells – which are then turbocharged through cellular engineering techniques that reprogram them 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 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 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 therapy.

Who can benefit from CAR-T therapy?

CAR-T 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.  Michigan Medicine also offers CAR-T therapy for adults through the Rogel Cancer Center.

What is CAR-T therapy like for the patient?

CAR-T therapy includes a number of steps.

  1. Evaluation: Patients referred to the C.S. Mott Children’s Hospital for relapsed or resistant leukemia treatment will be evaluated to see if they are eligible for the CAR-T 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 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 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 four weeks following the CAR-T infusion, so that the medical team can closely monitor for CAR-T 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, and fluid retention, changes in blood pressure and can require the patient be cared for in our pediatric intensive care unit (PICU) in severe instances. CRS typically develops within the first 1-2 weeks after the CAR-T 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 University of Michigan 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

The University of Michigan 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 therapy for adults with non-Hodgkin lymphoma.

The University of Michigan C.S. Mott Children's Hospital is the largest, most experienced pediatric marrow transplant 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 therapy at C.S. Mott Children’s Hospital, call 734-936-9814.