Early Results of Phase I Study of CAR T-Cell Therapy in Multiple Myeloma
Posted: Tuesday, January 15, 2019
LCAR-B38M, a bispecific chimeric antigen receptor (CAR) T-cell therapy targeting B-cell maturation antigen (BCMA), may prove to be an effective treatment of relapsed or refractory multiple myeloma, according to a phase I study presented at the 2018 American Society of Hematology (ASH) Annual Meeting & Exposition (Abstract 955). In this first-in-human study, Wan-Hong Zhao, MD, PhD, of The Second Affiliated Hospital of Xi’an Jiaotong University, Xi’an, China, and colleagues observed “deep and durable responses” among treated patients.
As of June 25, 2018, 57 patients have been infused with LCAR-B38M CAR T cells in this ongoing trial. Of those patients, 74% had stage III disease.
The overall response rate was 88%, after a median follow-up of 12 months. Of those responders, 42 patients achieved a complete response, 2 achieved a very good partial response, and 6 achieved a partial response. The median duration of response was 16 months; however, it was 22 months for those who achieved a complete response. The median progression-free survival was 15 months, but again, it was 24 months among those who achieved a complete response. There was no clear dose-response relationship, and BCMA expression did not seem to be associated with clinical response.
All treated patients experienced adverse events. The most common adverse events were pyrexia (91%), cytokine-release syndrome (90%), thrombocytopenia (49%), and leukopenia (47%). Overall, 65% of patients reported severe adverse events. During the study and follow-up period, 17 patients died of progressive disease (n = 14), suicide (n = 1), esophagitis (n = 1), and pulmonary embolism and acute coronary syndrome (n = 1).
A phase I/II study of LCAR-B38M in patients with relapsed or refractory multiple myeloma is underway in the United States.
Disclosure: The study authors’ disclosure information may be found at ash.confex.com.