A chimeric antigen receptor (CAR) T-cell therapy called idecabtagene vicleucel produces a complete response in at least a third of patients with relapsed and refractory multiple myeloma, according to results of the phase-2 KarMMA study. After a median of 13.3 months of follow-up, 73% of the 128 trial participants, who had received at least three previous regimens, responded to the treatment, with 33% having a complete response or better. Median progression-free survival was 8.8 months, researchers report in the New England Journal of Medicine. CAR-T therapy involves taking immune cells from a patient, engineering them to attack tumor cells and infusing them back into the patient. This specific treatment, also known as ide-cel or bb2121, was given a breakthrough-therapy designation by the U.S. Food and Drug Administration (FDA) in November of 2017. But in May the FDA declined to review the experimental treatment, saying it needed more details. "Ide-cel induced responses in a majority of heavily pretreated patients with refractory and relapsed myeloma," Dr. Nikhil C. Munshi of Dana-Farber Cancer Institute, in Boston, and colleagues write in their report, adding that 26% of treated patients became free of minimal residual disease. However, neutropenia was a problem in 91% of the volunteers, anemia in 70% and thrombocytopenia in 63%. One hundred seven patients developed cytokine release syndrome (84%); the syndrome was grade 3 or higher in seven patients. Bristol-Myers Squibb and bluebird bio Inc. announced preliminary results from the study in December 2019, listing the complete response rate as 31% after a median follow-up of 11.3 months. —Reuters Staff Source
