Who Might Benefit From CAR T-Cell Therapy?

Medically Reviewed by Michael W. Smith, MD on January 11, 2021

Chimeric antigen receptor (CAR) T-cell therapy is a new kind of cancer treatment. T cells from your immune system are changed in a lab and put back in your body to find and kill cancer cells.

This kind of therapy may work when other treatments haven’t. But it’s not right for everyone. Your doctor will think about your type of cancer, which treatments you've already had, and your health before recommending it for you.

Who Might Get CAR T-Cell Therapy?

CAR T-cell therapy is only approved to treat two groups of people with certain types of cancer:

  • Children and young adults up to age 25 with precursor B-cell acute lymphoblastic leukemia (ALL) that hasn't gotten better with treatment or that’s come back after treatment.
  • Adults with aggressive large B-cell lymphoma that hasn't gotten better with treatment or that’s come back after treatment.

"You have to have failed two prior therapies," says David Porter, MD, the Jodi Fisher Horowitz Professor in Leukemia Care Excellence and director of blood and marrow transplantation at the Hospital of the University of Pennsylvania. "Most of these patients have few, if any, effective treatment options available."

CAR T-Cell Therapy for Other Cancers

This treatment is still very new. Doctors have to learn more about it before they can use it earlier in the disease or to treat other types of cancer.

"You're using genetically modified human cells. You have to be very sure you understand when it works, who it works for, and also what the side effects are -- both short- and long-term," Porter says.

Doctors are learning more about CAR T-cell therapy by testing it in clinical trials. This is when researchers use new drugs or treatments with small groups of people to see how well they work.

Studies are looking to see if it might be right for other blood cancers including multiple myeloma and different forms of lymphoma and leukemia. Other studies are trying to find out if CAR T-cell therapy might work against solid tumors such as:

  • Lung cancer
  • Melanoma
  • Sarcoma
  • Ovarian cancer
  • Prostate cancer
  • Brain cancer

Taking part in a clinical study of CAR T-cell therapy could give you or your child the chance to try this treatment before it's approved for your cancer. But you have to find the right one.

"There are lots of resources for people who might be interested in looking into clinical trials," Porter says. "One of the best places to go is"

He also suggests that you ask your health care team to connect you with a clinical trial of CAR T-cell therapy in your area. Or check with an organization like the Leukemia & Lymphoma Society.

If there is a clinical trial for your type of cancer, you'll still need to qualify for it. The study's doctors will want to make sure you're healthy enough to benefit from the therapy.

When CAR T-Cell Therapy Might Not Be Right for You

There aren't any guidelines to keep you from getting this type of therapy if it's approved for your age group and your type of cancer. But because it can cause serious side effects, it may not be a good choice for people who aren’t in good health.

"Some patients will have things like heart disease or chronic kidney disease. We don't know if this treatment is safe in those situations," says Sattva Neelapu, MD, professor and deputy chair in the department of lymphoma/myeloma at The University of Texas M.D. Anderson Cancer Center.

"We have to evaluate those types of patients on a case-by-case basis to see which ones might qualify."

Show Sources


Dana-Farber Cancer Institute: "CAR T-Cell Therapy: Is It Right for You?" "CAR T-Cell Therapy Gives Cancer Patients New Hope."

Leukemia & Lymphoma Society: "Chimeric Antigen Receptor (CAR) T-Cell Therapy."

National Cancer Institute: "CAR T-Cell Therapy Approved for Some Children and Young Adults with Leukemia."

Sattva Neelapu, MD, professor, deputy chair, department of lymphoma/myeloma, division of cancer medicine, The University of Texas M.D. Anderson Cancer Center.

David Porter, MD, the Jodi Fisher Horowitz Professor in Leukemia Care Excellence; director of blood and marrow transplantation, Hospital of the University of Pennsylvania.

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