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Gene-Therapy Approval May be Cancer Game Changer

FDA ruling on leukemia treatment may impact adult cancers as well

Gene therapy to fight cancer

Andrew Brookes/Getty Images

Researchers are exploring using a patient's own T cells to fight cancer.

A new ruling from the Food and Drug Administration (FDA) may transform the future of cancer treatment by approving the first-ever treatment that genetically alters a patient’s own cells to fight cancer. 

The new therapy, which is marketed as Kymriah by the company Novartis, is a customized treatment that's created using an individual patient’s own T cells, a type of white blood cell. The patient’s T cells are collected and then genetically modified to include a new gene with a specific protein called a chimeric antigen receptor (CAR) that directs the T cells to target and kill specific leukemia cells. Once modified, the cells are infused back into the patient to kill the cancer cells.



While this FDA approval only applies to a gene therapy drug for children and young adults with an aggressive type of leukemia, researchers across the industry are working to develop gene therapies for other types of cancers, including those impacting older adults. In remarks accompanying the announcement, FDA Commissioner Scott Gottlieb said that more than 550 types of experimental gene therapies were being studied.

Kymriah ushers in “a new approach to the treatment of cancer and other serious and life-threatening diseases,” the FDA said in a statement, noting that the new therapy is “the first gene therapy available in the United States.”

The drug is not without its complications, including dangerous side effects such as severe drops in blood pressure and risk for infection. The FDA requires that hospitals and doctors be specially trained, certified and equipped to administer the drug. It is also a very costly treatment, at $475,000. 

Learning Curve Ahead

While the approval of this particular medication does not have much application for older people, the medical approach that this represents will "have a broader impact on adult cancers, including lymphoma and other forms of leukemia," Len Lichtenfeld, deputy chief medical officer for the American Cancer Society, tells AARP. Researchers are looking at the CAR-T approach for treatment of solid tumors as well as for myeloma, a blood cancer that is more common in older adults, Lichtenfeld says, although that research is not as far along. 

As these types of CAR-T treatments are put into practice, it is important for people to be aware that not every patient will respond in the same way to treatment. Given the long-term and possibly life-threatening side effects, it might not be the ideal treatment for everyone, and age could play a factor in utilization.

"There is still much that we must learn," Licthenfeld says. "Where will CAR-T fit in the treatment program looking forward? Will this be a treatment that will be a last resort or used earlier in the course of any one person’s illness? It is going to take us a while to not only understand the treatments, but to understand who will benefit the most and where it fits in the treatment program."

Lichtenfeld is also hopeful that Medicare will cover the treatment when it is available for older adults. "Although treatment is expensive, based on past experience I think Medicare will cover it," he says.

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