# CARTITUDE-1: cilta-cel, a BCMA CAR-T, in heavily pretreated myeloma

Source: https://onco.cc/key-papers/paper-cartitude-1-cilta-cel-lancet-2021/  
OnCo record `paper-cartitude-1-cilta-cel-lancet-2021` (Key paper). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

A single infusion of BCMA-directed CAR-T cells produced responses in 97% of patients whose myeloma had failed a median of six prior lines, with two-thirds reaching stringent complete response.

## Summary

CARTITUDE-1 was a phase 1b/2 single-arm study of ciltacabtagene autoleucel (cilta-cel), a CAR-T with two BCMA-binding domains, in 97 patients with relapsed or refractory multiple myeloma exposed to a proteasome inhibitor, an immunomodulatory drug and an anti-CD38 antibody (median six prior lines, 88% triple-class refractory). The overall response rate was 97%, with stringent complete response in 67%; 12-month PFS was 77% and OS 89%. Cytokine release syndrome occurred in 95% (grade 3-4 in about 4%) and neurotoxicity in about 21%, including a small number of delayed movement and neurocognitive events. Long-term follow-up showed roughly a third of patients progression-free at five years without further therapy, unprecedented in this population.

## Fields

- Kind: Key paper
- Last checked: 2026-09-08
- Journal: The Lancet
- Year: 2021
- DOI: 10.1016/S0140-6736(21)00933-8
- Authors: Berdeja JG, Madduri D, Usmani SZ, et al.
- Findings: 97 patients infused; median 6 prior lines; 88% triple-class refractory.; Overall response 97%; stringent complete response 67%; MRD-negativity in most evaluable responders.; 12-month PFS 77%, OS 89%; median PFS about 35 months in later follow-up; about a third progression-free at 5 years.; CRS in 95% (grade 3-4 about 4%); ICANS 17%; delayed movement/neurocognitive syndrome in a minority.; Median time to CRS onset was 7 days, later than with CD19 CAR-Ts, enabling outpatient monitoring strategies.
- What it means: CARTITUDE-1 showed that a single CAR-T infusion can put late-stage myeloma into deep, multi-year remission, leading to FDA approval of cilta-cel in 2022 for heavily pretreated disease. It set the efficacy bar for BCMA-directed therapy and motivated moving CAR-T earlier (CARTITUDE-4). Late neurological toxicity and secondary malignancies remain the safety questions.
- Caveats: Single-arm trial in fit, selected patients; no randomised comparator.; Manufacturing failures and bridging deaths are not captured in infused-patient analyses.; Delayed parkinsonism-like neurotoxicity and rare second primary malignancies (including T-cell lymphoma) emerged with follow-up.; Access limited by manufacturing slots and cost.

## Sources

- PubMed search: https://pubmed.ncbi.nlm.nih.gov/?term=CARTITUDE-1%20ciltacabtagene%20autoleucel%20Berdeja%20Lancet%202021
- ClinicalTrials.gov NCT03548207: https://clinicaltrials.gov/study/NCT03548207

## Connected records

- key papers: [CARTITUDE-4: cilta-cel CAR-T versus standard combinations after one to three prior lines of myeloma therapy](https://onco.cc/key-papers/paper-cartitude-4-cilta-cel-nejm-2023/)
- cancers: [Multiple myeloma](https://onco.cc/cancers/multiple-myeloma/)
- technologies: [CAR-T cell therapy](https://onco.cc/technologies/car-t/)
- targets: [BCMA](https://onco.cc/targets/bcma/)
- drugs: [Ciltacabtagene autoleucel](https://onco.cc/drugs/ciltacabtagene-autoleucel/)
- companies: [Johnson & Johnson](https://onco.cc/companies/johnson-johnson/), [Legend Biotech](https://onco.cc/companies/legend-biotech/)
- terms: [Cytokine release syndrome (CRS)](https://onco.cc/terms/crs/), [ICANS (neurotoxicity)](https://onco.cc/terms/icans/), [Objective response rate (ORR)](https://onco.cc/terms/orr/)
- trials: [CARTITUDE-1](https://onco.cc/trials/cartitude-1/)
- people: [Jesús G. Berdeja](https://onco.cc/people/jesus-berdeja/), [Saad Z. Usmani](https://onco.cc/people/saad-usmani/)
- bottlenecks: [Manufacturing cost and time for living and radioactive medicines](https://onco.cc/bottlenecks/b-manufacturing-cell-therapy/), [Toxicity and quality of life are undervalued](https://onco.cc/bottlenecks/b-toxicity-qol/)
- journals: [The Lancet](https://onco.cc/journals/lancet/)

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