Pediatric Leukemias: CAR T Cell Therapy for Children, Teens, and Young Adults

Chimeric antigen receptor (CAR) T cell therapy is a promising new treatment that redirects the body’s immune system to fight cancer. The power behind this therapy is the body’s own T cells, which normally fight viruses and bacteria and are one of the body’s strongest lines of defense.

CARs enable T cells to recognize and kill cancers like leukemia. This remarkable therapy is sometimes referred to as a “living drug” because it is made from your child’s own cells. CAR T cells can remain inside the body, protecting against cancer for months and potentially years.

Pediatric oncologist Kevin Curran with CAR T patient Esmeralda Pineda
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Here’s how it works: The patient’s T cells are collected from his or her blood. In a lab, scientists insert a gene into the T cells that enables them to make the CAR protein. The transformed T cells are then infused back into the patient, where they can now recognize cancer and kill it.

In clinical trials, this therapy has shown it can kill the most common cancer in children and young adults, acute lymphoblastic leukemia (ALL).

To learn more about CAR T cell therapy, view our frequently asked questions below.

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1. Is my child eligible for CAR T cell therapy?

The US Food and Drug Administration has approved the use of CAR T cell therapy for patients with B cell acute lymphoblastic leukemia (B-ALL) up to age 25 whose cancer has not responded to other treatments or has returned (relapsed).

CAR T cell therapy for other cancers such as lymphoma is under review by the FDA. MSK is conducting clinical trials of CAR T cell therapy for other blood and solid-tumor cancers. You can learn more about open clinical trials at MSK using CAR T cells.

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2. Is CAR T cell therapy right for my child?

MSK is committed to finding the right treatment strategy for your child, which may include the use of CAR T cells. Please call 1-888-MSK-CART, and a member of our CAR T staff will speak with you about your child. If CAR T therapy is the right choice for your child, our goal is to have him or her seen by our team of CAR T specialists as soon as possible. We have a dedicated team to help coordinate your appointment and are committed to offering your child the best chance of success.

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3. Who will treat my child?

MSK has a multidisciplinary team at the ready for you and your family. They have been treating patients since the inception of this breakthrough therapy. The team includes specialists in bone marrow transplantation, neurology, and intensive care, and nurses with specialized training in caring for children and young adults being treated with CAR T cell therapy.

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4. What are the first steps for CAR T cell therapy?

Learn why CAR T cell therapy is the best form of treatment for some pediatric leukemias.

After your phone call or referral, we will request your child’s medical records. We will consider your child’s prior treatment, any complications from treatment, and information on the type of cancer and the current status of your child’s disease. Next, we will schedule an initial consultation with one of our CAR T cell team members. If we decide together that CAR T cell therapy is appropriate, we’ll schedule a collection (called leukapheresis) of your child’s T cells. Most children will require a specialized catheter (tube) placed prior to collection of T cells.

Once collected, your child’s T cells will be sent to a lab for genetic modification to create CAR T cells. While we wait for the CAR T cells, we may give your child chemotherapy to reduce the amount of leukemia in the body. We will try to do this on an outpatient basis so your child won’t have to stay overnight in the hospital and can even return home for treatment, if possible.

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5. What happens when it’s time for my child to receive the new T cells?

It takes 20 to 30 days for the CAR T cells to be ready for infusion. Before the infusion, we will give your child a cycle of chemotherapy called conditioning chemotherapy, which will improve the ability of CAR T cells to work within the body. Two to 14 days after completion of the chemotherapy, we will infuse the CAR T cells back into the body. This takes about 30 minutes and causes minimal side effects during or immediately after the infusion.

Conditioning chemotherapy and infusion of CAR T cells can be done as an outpatient or inpatient. This depends on the status of your child’s leukemia and overall health.

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6. Will my child experience any side effects from CAR T cell therapy?

Side effects following infusion of CAR T cells can be mild to severe. Before the infusion, our MSK CAR T cell team will discuss what side effects are expected after the procedure. Most side effects occur when the CAR T cells grow in the body and begin attacking cancer cells. This process results in the release of cytokine proteins, which can cause flulike symptoms that can be mild to severe. They include fever, rapid heart rate, low blood pressure, low oxygen level, or difficulty breathing. Other side effects following CAR T cell infusion can include decreased kidney function and possible neurologic changes like confusion, sleepiness, and seizures.

MSK’s team of CAR T cell experts — some of the most experienced in the world when it comes to CAR T therapy — are dedicated to monitoring and managing any side effects that come about as a result of this pioneering new treatment. It is important to understand that your child does not need to suffer severe side effects. Many of the children and young adults treated at MSK with CAR T cell therapy have had minimal side effects and a good response to the therapy.

Pediatric oncologist Kevin Curran answers five questions about CAR T cell therapy.
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7. How long will my child stay in the hospital?

The length of hospitalization for your child will depend on the severity of his or her cancer and his or her response to the CAR T therapy. Some patients may need up to 30 days of in-hospital monitoring after their T cells are given back to them to manage any side effects. Some patients will require less time.

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8. Are there resources to help families who need to travel for this therapy?

Families from around the United States and the world come to MSK to have their children treated with CAR T cell therapy. MSK has many resources available to help families who come to us for treatment, including local housing and transportation. The CAR T cell team will connect you with members of our staff who can assist with travel and other logistics.

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9. Will this therapy cure my child’s leukemia?

The ultimate goal of CAR T cell therapy is the “definitive” treatment of leukemia that resists other treatments or has returned. Following treatment, we will monitor your child’s blood and bone marrow to look for evidence of cancer and to see if the CAR T cells are still functional. We will watch what the CAR T cells are doing in the body to make sure the cells are still active and fighting cancer for months and even years after the treatment.

Any treatment with gene therapy will require us to monitor your child for up to 15 years. The first five years will include blood tests to make sure your child is cancer free. The next ten years will include regular check-ins to make sure he or she remains healthy.

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10. Does this therapy sometimes fail?

The majority of patients treated with CAR T cells for B-ALL have responded successfully following infusion. However, CAR T cell therapy is a highly personalized treatment, and it has failed for some patients. Additionally, for some patients, their leukemia has returned following an initial period of remission. MSK’s CAR T cell team is committed to finding the best treatment options for your child.

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11. How much will this cost? Will insurers or the government cover it?

Our CAR T coordinators will connect you with one of our trained financial advisors to answer any questions you may have.

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12. Why should I bring my child to MSK to receive CAR T cell therapy?

MSK is one of the most experienced centers in the world in the use of CAR T cell therapy. From that experience, we’ve developed a highly skilled team that can manage side effects and provide your child with the best possible care and chance of cure. Much of the pioneering work on CAR T therapy was conducted at MSK. We continue to make innovations in the research and use of these therapies.

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