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CAR T-Cell Treatment for Leukemia

Engineering a Smarter, Stronger Immune System

The immune system is your body’s security system. It fights off anything that doesn’t belong, namely, viruses, bacteria, fungi and toxins.

White blood cells are an essential part of the immune system. They form in your bone marrow (the spongy material inside bones). One type of white blood cell is called a lymphocyte. There are two types of lymphocytes:

  • B-cells: B-cells are the “brains” of the operation. They find invading enemies and send in reinforcements — the T-cells — to stop them.
  • T-cells: The T-cells are the “muscle.” They destroy the targets that the B-cells found.

In acute lymphoblastic leukemia (ALL), cancer affects how these lymphocytes develop so they can’t do their job right. But with CAR T-cell therapy for leukemia, doctors reprogram the healthy T-cells to kill leukemia cells.

How CAR T-Cell Treatment for Leukemia Works

CAR T-cell therapy is a revolutionary new treatment for leukemia. It is a combination of gene therapy and immunotherapy.

During leukemia CAR T-cell therapy:

  1. Doctors draw your child’s blood to get their T-cells.
  2. Using gene therapy, they reprogram the T-cells to make the CAR protein. The CAR protein makes it possible for them to recognize and attack only cancer cells. This process takes about a month.
  3. Doctors then multiply the changed T-cells in a laboratory.
  4. Some patients get chemotherapy while waiting for their new T-cells. The chemotherapy lessens the amount of leukemia in their body to prepare it for the new cells.
  5. When there are enough T-cells, doctors give the changed cells back to your child intravenously (through an IV).

Once in your child’s body, the new T-cells grow in number and get rid of the cancer. And because these cells stay in your child’s body for months, maybe years, they may provide long-term protection.

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T-Cell Treatment for Leukemia at Nemours Children’s: Why Choose Us

Our doctors have special expertise in this breakthrough therapy. For example, Nemours Children’s Hospital, Delaware is among a small group of hospitals designated as a CAR-T Center. We earned this designation in 2018 because of our:

Children in Florida can get this lifesaving treatment at Nemours Children’s Health, Jacksonville and our partner Wolfson’s Children's Hospital in Jacksonville. The Ronald McDonald House of Delaware and Ronald McDonald House of Jacksonville are nearby for families that need a place to stay during treatment.

A transplant coordinator supports and guides you from the start. Your coordinator is a familiar face throughout the process, on-hand to:

  • Help your family understand what to expect at each stage of treatment, answer questions and address concerns
  • Arrange and coordinate appointments
  • Prepare you for life at home after treatment

Find Leukemia Care From Nemours

We have many pediatric leukemia experts and locations — find the care closest to you:

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CAR T-Cell Therapy vs. Bone Marrow Transplant

Both bone marrow transplants (also known as stem cell transplants) and CAR T-cell therapy effectively treat childhood leukemia. But there are some differences.

Doctors can use bone marrow transplants to treat several types of childhood leukemia, including ALL, acute myeloid leukemia (AML) and chronic myeloid leukemia (CML). Currently, doctors can only use CAR T-cell therapy to treat B-cell ALL. B-cell ALL is the most common type of leukemia that relapses.

CAR T-cell therapy tends to have fewer side effects than stem cell transplants because:

  • Kids receive lower doses of chemotherapy.
  • It involves the patient’s own cells — not a donor’s. Using your child’s own cells lowers the risk for complications like graft-versus-host disease, where the immune system attacks the patient's healthy cells.

Who Can Get Leukemia CAR T-Cell Therapy?

Because of ongoing leukemia clinical trials, the list of children who can get CAR T-cell therapy grows each year. Children with other kinds of leukemia or lymphoma may also be able to get this therapy in the future as we continue to prove its safety and effectiveness.

In 2017, the FDA approved the use of CAR T-cell therapy in children with B-cell ALL that has relapsed. If your child has been diagnosed with B-cell ALL that has relapsed or not responded to other treatments, they may be eligible for CAR T-cell therapy.