At the recent American Society of Clinical Oncology (ASCO) annual meeting, researchers unveiled remarkable long-term results for ciltacabtagene autoleucel (cilta-cel; Carvykti), a CAR T-cell therapy, in patients with heavily pretreated relapsed or refractory multiple myeloma. The findings from a post-hoc analysis of the CARTITUDE-1 trial suggest that, for some, a single infusion of cilta-cel may offer not just extended survival but the potential for a functional cure.

Impressive Long-Term Survival

First reported by Medpage Today, the study followed 97 patients who had exhausted at least three prior lines of therapy and were considered triple-class refractory—a group typically facing a median overall survival (OS) of just one year. In contrast, patients treated with cilta-cel achieved a median OS of 60.7 months, with 45 individuals still alive at long-term follow-up. Notably, 32 patients remained alive and progression-free after five years, without any additional anti-myeloma treatment or maintenance therapy.

Among a subset of 12 patients assessed for minimal residual disease (MRD), all were MRD-negative and imaging-negative at least five years after their single infusion, suggesting either a durable remission or, as investigators cautiously propose, the first signs of a true cure in this disease setting.

A Plateau in Survival Curves

One of the most striking aspects of the analysis is the apparent plateau in progression-free survival (PFS) curves at around 60 months. This plateau hints at a group of patients—about one-third—who experience sustained benefit and potentially long-term freedom from disease. Dr. Krina Patel, an ASCO discussant, called this the first evidence of a possible cure for relapsed/refractory multiple myeloma in the modern treatment era.

Broad Applicability and Durable Responses

The study population was diverse, including patients with high-risk cytogenetics and extramedullary disease. Those who remained progression-free at five years generally had a lower initial tumor burden, but durable remissions were also observed in patients with more aggressive disease features. This suggests the therapy’s potential extends beyond standard-risk patients.

A New Standard Emerges

The robust results from CARTITUDE-1 were the basis for cilta-cel’s FDA approval in relapsed/refractory myeloma after four or more prior therapies. Subsequent studies, like CARTITUDE-4, have led to expanded approvals, allowing more patients access to this innovative approach.

Looking Forward

While some patients may eventually relapse, the unprecedented durability of response and the emergence of a long-term progression-free plateau mark a turning point in multiple myeloma treatment. For patients and clinicians, these results bring hope that a single, personalized CAR T-cell infusion could offer lasting remission, and for some, perhaps a cure.

In conclusion, cilta-cel’s performance in the CARTITUDE-1 trial redefines expectations for advanced multiple myeloma, offering a new era of hope for those with few remaining options.