Six Things Parents Need to Know About CAR T-Cell Therapy for Cancer

What happens when cancer doesn’t respond to chemotherapy?  

A revolutionary therapy called chimeric antigen receptor (CAR) T-cell therapy uses a person’s own immune cells that have been modified to recognize and attack cancer cells. In 2018, Children’s Hospital Los Angeles became the first hospital in Southern California approved to deliver this new therapy to pediatric patients with acute lymphoblastic leukemia who did not respond to other treatments.

Since then, the hospital has treated more than 140 patients with CAR T-cell therapy, now approved to treat a variety of cancers in patients who have stopped responding to chemotherapy. CHLA is also conducting clinical trials to further advance this therapy for some of the most aggressive blood, brain, and solid tumor cancers, as well as autoimmune diseases.  

“CAR T-cell therapy is transforming cancer treatment by harnessing the patient’s own immune system as a powerful tool against the disease,” says Paibel Aguayo-Hiraldo, MD, Medical Director of Transplantation and Cellular Therapy at CHLA. “As we refine this therapy and expand its applications, we are not just treating cancer—we are redefining the possibilities of medicine.”

Here are six key things to know about CAR T-cell therapy.

1. It may be life-changing for kids with relapsed acute lymphoblastic leukemia (ALL).

Previously, patients whose disease wasn’t responding to chemotherapy, or who relapsed after receiving a bone marrow transplant, had limited treatment options that offered the potential for a cure. CAR T-cell therapy has been shown to control leukemia in the large majority of these patients, with more than half remaining in remission 1 year later.

2. CAR T-cell therapy is a type of cell and gene therapy.

CAR T-cells are designed to provide a lifetime of protection in case cancer cells re-emerge. Following therapy, your doctor will monitor your child’s blood and bone marrow to make sure the modified T-cells are still active and that there is no remaining cancer.  

Since CAR T-cell therapy is considered a type of cell and gene therapy, your child will need to be monitored for 15 years after treatment. The follow up period includes five years of close monitoring to determine that the CAR T-cells are still effective and that your child is cancer-free. For the following ten years, your child will have an annual check-in that may include a blood test to ensure they remain healthy. The follow-up testing may change if your child needs a different type of cell therapy; for example, a bone marrow transplant.

3. CHLA doctors are trialing expanded use cases for CAR-T cell therapy.  

“Given the response to CAR T-cell therapy that many of our ALL patients have experienced, our doctors are eager to extend this treatment to patients with other cancers, and even other diseases such as the autoimmune disease lupus, when conventional therapies are not adequate,” says Sarah Richman, MD, PhD, Attending Physician in the Cancer and Blood Disease Institute.  

CHLA is currently enrolling patients on a trial of CAR T-cell therapy for brain tumors and anticipates enrolling patients with certain types of solid tumors, and will soon begin enrolling patients with lupus.

To find a clinical trial at CHLA, visit our Research Study Database or speak with your CHLA specialist or your pediatrician.

4. CAR T-cell therapy may have side effects.

All cancer treatments have side effects. Within three to ten days of infusion, a patient receiving CAR T-cells may develop flu-like symptoms that can be quite severe. To help manage their symptoms, most children need to be hospitalized and some need to be treated in the intensive care unit.

5. It takes a few weeks to begin treatment.

If you and your child’s doctor determine that CAR T-cell therapy is the best option, the next step will be to collect T-cells from your child. Cell collection may require that your child be fitted with a special device called a catheter. After collection, the cells are frozen and shipped to a lab where they are engineered to seek out and destroy cancer cells. The cells are then returned to CHLA for your child to begin treatment.  

This process may take a few weeks. If your child has aggressive cancer, they may need other therapies to keep the cancer under control while they wait to begin treatment.

6. Doctors at Children’s Hospital Los Angeles are experts at delivering CAR T-cell therapy.

CHLA doctors helped develop this treatment by conducting clinical trials to test CAR T-cell therapy before it was approved by the U.S. Food and Drug Administration. Later, the hospital was among the first institutions approved to deliver CAR T-cell therapy to children, teenagers, and young adults.  

Today, CHLA has established a team of specialists—including oncologists, hematologists, nurses and psychologists—with a wealth of expertise in delivering CAR T-cell therapy and providing the best overall care for your child.