What is CAR T-Cell Therapy?

CAR T-cell therapy (also called chimeric antigen receptor T-cell therapy) is a type of cancer treatment. It uses infection-fighting cells in the immune system (T cells) to detect and destroy cancer cells.

Who Can Benefit from CAR T-Cell Therapy?

The Food and Drug Administration has approved CAR T-cell therapy for patients with relapsed and treatment-resistant B-cell blood cancers who haven’t responded well to other cancer treatments.

These cancers include:

• B-cell acute lymphoblastic leukemia (B-ALL) for patients up to age 26 (second relapse or later)
• Diffuse large B-cell lymphoma for patients older than 18

Clinical trials using CAR T cells are also available for people with some other forms of blood cancer.

CAR T-Cell Therapy Treatment Steps

CAR T-cell therapy happens in the hospital. It is an inpatient treatment. Patients should expect to be in the hospital for up to four weeks for treatment and observation.

Treatment steps include:

• Collection: The care team collects some white blood cells (including T-cells) from the patient.
• Modification: The care team sends the collected cells to an outside laboratory. In the lab, the T-cells are taught to grow special receptors on their surface, called chimeric antigen receptors (CAR) that help T-cells spot and attach to cancer cells.
• Multiplication: The new CAR T-cells multiply in the lab, are frozen, and are then returned to the hospital.
• Chemotherapy: Patients receive conditioning chemotherapy (lymphodepletion) a few days before treatment to make room for the CAR T cells to work.
• Infusion: The CAR T-cells are infused into the patient’s blood in one infusion.

The CAR T-cells target a specific protein called CD19 that is on the surface of B-cell cancers. If all goes as planned, the CAR T-cells continue to multiply in the patient's body and kill cancer cells that have the CD19 protein on their surfaces.

Finding a Hospital That Provides CAR T-Cell Therapy

CAR T-cell therapy is a highly specialized and personalized treatment that is only available at a small number of hospitals. Cincinnati Children’s has extensive CAR T-cell therapy experience. It was one of the first pediatric hospitals to offer CAR T-cell therapy clinical trials.

CAR T-Cell Therapy Benefits

Until recently, patients with relapsed or treatment-resistant B-cell blood cancers had very few treatment options. CAR T-cell therapy offers new hope. For example, in clinical studies, 62% of patients who had the CAR T-cell therapy using a drug called KYMRIAH® were in remission two years post-treatment.

CAR T-cell therapy can be a game changer when standard treatment is not working. But it isn’t for everyone. The doctor will discuss the options with you before making a recommendation about whether this therapy or another advanced cancer treatment is the best option.

CAR T-Cell Therapy Risks

CAR T-cell therapy may have side effects. Some side effects can be serious but usually go away in a few days without long-term problems. These side effects include cytokine release syndrome (CRS), which causes flu-like symptoms and neurologic problems like confusion. A hospital stay is necessary to allow the care team to watch patients for serious side effects and provide early treatment if needed. About 30% of patients who have CAR T-cell therapy are admitted to the intensive care unit to be more closely monitored.

CAR T-Cell Therapy Recovery

The recovery time for CAR T-cell therapy is much shorter and less intense than it is for a bone marrow transplant. Most patients return to their normal lives within a month or two. A doctor will continue to monitor them for side effects and treatment response.

Many patients need long-term immunoglobulin replacement therapy (infusions) after CAR T-cell therapy to help boost their immune system.