A drug called Axicabtagene ciloleucel (KTE-C19) has become the first chimeric antigen receptor (CAR) T-cell therapy approved to treat a form of cancer in adults, the Food and Drug Administration (FDA) announced today.
The decision means the drug, known as Yescarta, can now be used for some adults with refractory aggressive non-Hodgkin lymphoma (NHL). The FDA ruling in favor of KTE-C19 is based on the results of a series of clinical trials that showed the therapy to be safe and effective. In a trial featuring 101 patients, 82 percent of patients responded to the treatment, and 54 percent of patients had a complete response to therapy, according to results presented in June. Thirty-six percent of patients remain in complete remission six months after treatment.
“Treating patients with CAR T cells has been one of my most exciting professional experiences, and the FDA approval of this therapy offers hope and optimism to a subset of patients whose other treatments have failed them,” says Caron A. Jacobson, MD, Medical Director of the Immune Effector Cell Therapy program at Dana-Farber/Brigham and Women’s Cancer Center (DF/BWCC). “It is extremely rewarding to be able to offer a new therapy to patients who had virtually no other options just 12 to 24 months ago.”
The patients Jacobson and her team are currently treating with CAR T-cell therapy at DF/BWCC have highly refractory and fast-growing blood cancers, and have suffered a year or more of disappointing results with relatively toxic therapies.
“This therapy requires just a one-time infusion for patients, and the results are evident within one month,” Jacobson says. “It is our goal as clinicians to help patients and improve their quality of life. Seeing these patients return to work, their families and their livelihoods so quickly is an important reminder of how far we have come. It is also inspiration for the work we still need to do.”
The approval follows the FDA’s recent first-ever approval of CAR T-cell therapy, in August, for the treatment of some pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL).
CAR T-cell therapy, like all forms of cancer immunotherapy, seeks to sharpen and strengthen the immune system’s inherent cancer-fighting powers. It involves treating patients with modified versions of their own immune system T cells – white blood cells that help protect the body from disease.
To convert normal T cells into CAR T cells, technicians first extract T cells from a patient’s blood and genetically engineer them in a lab to produce proteins on their surface called chimeric antigen receptors, or CARs. The CARs serve a dual purpose: to enable the T cells to latch onto specific tumor cell proteins called antigens, and to signal the T cells to kill those tumor cells. The newly minted CAR T cells are allowed to reproduce in a lab until they number in the hundreds of millions, and they’re infused into the patient. If all goes as planned, the CAR T cells will continue to reproduce and serve as an effective fighting force against cancer cells.
The initial clinical trials of CAR T cell therapy have involved pediatric and adult patients with blood-based cancers such as leukemia, lymphoma, and multiple myeloma. Based on the therapy’s striking success in many patients, CAR T-cell therapy trials are now opening for certain types of solid tumors as well.