A New CAR-T Cell Therapy for Advanced B-cell Acute Lymphoblastic Leukemia  

THE FDA APPROVED THE FIRST CAR-T CELL THERAPY FOR CERTAIN PATIENTS WITH this aggressive form of LEUKEMIA  

The U.S. Food and Drug Administration (FDA) approved brexucabtagene autoleucel (Tecartus) for certain adult patients with B-cell acute lymphoblastic leukemia (ALL) that came back or did not respond to prior treatment.  

Brexucabtagene autoleucel is a chimeric antigen receptor (CAR-T) cell therapy, a type of treatment in which a patient’s T cells are engineered in the laboratory to produce molecules that allow them to recognize and attack cancer cells. The engineered T cells are grown to large numbers and given to the patient by infusion. In this case, the CAR T cells recognize the CD19 protein found on most B cell malignancies. 

Acute Lymphoblastic Leukemia (ALL) cells
Image via Wikimedia.

The FDA approval is for adults with relapsed or refractory B-cell precursor ALL, the most common form of ALL. This aggressive cancer affects immature white blood cells called B-cell lymphoblasts in the blood and bone marrow. Brexucabtagene autoleucel is the first FDA-approved CAR-T cell therapy for this patient population. 

The efficacy of brexucabtagene autoleucel was evaluated in a single-arm, multicenter clinical trial. Patients received a single infusion after completing a conditioning regimen of chemotherapy to deplete their immune cells and make room for the CAR T cells. Among 54 patients, 28 (52 percent) experienced a complete response within three months of infusion. The median follow-up for patients who responded was 7.1 months. The median duration of complete response was not reached; however, it was estimated to be greater than 12 months for more than half of the study participants. 

The approval came with a boxed warning to advise health care professionals of the risks of cytokine release syndrome, an acute inflammatory syndrome with fever and multiple organ dysfunction, and neurologic toxicities. 

It was estimated that 5,690 adults and children would be diagnosed with ALL in the U.S. in 2021.  

The FDA decision was rendered on October 1, 2021.