Idecabtagene vicleucel（ide-cel）CAR-T 细胞治疗复发/难治性多发性骨髓瘤

Idecabtagene vicleucel (ide-cel), a novel chimeric antigen receptor (CAR) T-cell therapy targeting B-cell maturation antigen (BCMA), has recently gained approval by the US FDA for relapsed and refractory multiple myeloma (RRMM) after multicenter trials have demonstrated unprecedented results in this difficult-to-treat subgroup of patients. As the first CAR T-cell product approved for myeloma, ide-cel is poised to become a practice-changing treatment option. This first-in-class therapeutic offers hope for more durable remissions, as well as better quality of life, following a single infusion in a group of patients that previously had little hope. This paper reviews the ide-cel product in terms of design, pharmacology, efficacy and toxicity as described in studies reported to date.

Lay abstract Idecabtagene vicleucel (ide-cel) is a novel therapy using a patient s own T cells that have been genetically modified to force them to recognize a target antigen called BCMA that is present on myeloma and plasma cells but not on other normal cells. This therapy, known as CAR T-cell therapy, has recently gained approval by the US FDA for relapsed multiple myeloma after clinical trials have demonstrated unprecedented results in this difficult-to-treat subgroup of patients who have failed at least four prior lines of therapy.

As the first CAR T-cell product approved for myeloma, ide-cel is poised to become a practice-changing treatment option. This first-in-class therapeutic offers hope for more durable remissions, as well as a longer and better quality of life, following a single infusion. This paper reviews the ide-cel product in terms of design, pharmacology, efficacy and toxicity as described in studies reported to date.