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FDA Reviews CAR T-Cell Therapy: What You Need to Know

3 min read  |  December 15, 2023  | 

Over the past decade, researchers have taken historic steps toward developing a cancer therapy using “CAR T cells.” This therapy using genetically engineered T-cells from your immune system (the CAR stands for chimeric antigen receptors), holds the potential to revolutionize the way doctors treat a wide array of blood cancers. But last month, the U.S. Food and Drug Administration (FDA) announced it would conduct a review of those therapies after receiving reports of patients developing secondary cancers from the treatments.

The news surprised the medical community and sent stock prices plunging for the six pharmaceutical companies that manufacture CAR T cells. Leaders at Sylvester Comprehensive Cancer Center, part of the University of Miami Health System, however, caution that the federal review is a customary step during the evaluation of any cancer therapy.

Risk of secondary cancer is low

The FDA ordered its review after receiving a total of 20 reports of secondary cancers. This represents a tiny percentage of cases considering that CAR T-cell therapy has been administered to tens of thousands of patients, according to Mikkael Sekeres, M.D., Chief of the Division of Hematology at Sylvester and a professor at the University of Miami Miller School of Medicine. 

“It’s a ratio of about one in a thousand,” Dr. Sekeres says. “Any therapy that we offer to fight cancer has risks. But if I were considering CAR T-cell therapy, it would not be a stumbling block to move forward and receive that therapy because the risk is just so low.”

The FDA echoed that sentiment: “The overall benefits of these products continue to outweigh their potential risks for their approved uses.” 

How CAR T-cell therapies work in treating cancer

The human body naturally produces immune cells called T cells that attach themselves to foreign antigens and trigger other parts of the immune system to destroy the foreign substance. CAR T-cell therapies work by removing those T cells from a patient’s blood and genetically altering them in a lab. The T cells are modified to target specific cancer cells and reinserted into the patient’s bloodstream.

This technology has allowed doctors to finetune CAR T cells to target different types of cancer. CAR T-cell immunotherapy has revolutionized cancer treatment and has been initially effective in leukemia and lymphoma, with some patients remaining in remission. Given its potential, researchers worldwide are working to expand CAR T-cell therapy to a broader range of cancers.

Navigating the risks and benefits

Cancer treatments undergo ongoing reviews to evaluate efficacy, success, and potential side effects. From radiation to chemotherapy, all cancer treatments have been closely scrutinized with multiple levels of clinical trials before they are widely available to patients.

While doctors at Sylvester have been administering CAR T-cell treatments for several years, Dr. Sekeres says they are taking the FDA review seriously. The team has already incorporated the new risk into their risk-benefit discussions with patients. But he says they intend to continue using CAR T-cell therapy. The treatment is a valuable tool in their arsenal and holds future potential as it’s tested, evaluated, and improved. 

“It offers an effective therapeutic option to people who previously had none,” Dr. Sekeres says. “And in some patients, we suspect it may even be curative.”


Alan Gomez is a contributor to the UHealth Collective news site.


Tags: cancer treatment, car t-cell therapy, clinical trials, Dr. Mikkael Sekeres, fda review, leukemia treatment

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