# ZUMA-1: axicabtagene ciloleucel for refractory large B-cell lymphoma, the first CAR-T approved for lymphoma

Source: https://onco.cc/key-papers/paper-zuma-1-axi-cel-nejm-2017/  
OnCo record `paper-zuma-1-axi-cel-nejm-2017` (Key paper). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

In lymphoma refractory to chemotherapy, where expected survival was around six months, a single CAR-T infusion produced responses in 82% of patients and long-term remission in about 40%.

## Summary

ZUMA-1 was a multicentre single-arm phase 2 trial of axicabtagene ciloleucel (axi-cel), a CD19 CAR-T with a CD28 costimulatory domain, in 111 enrolled and 101 treated patients with refractory diffuse large B-cell, primary mediastinal or transformed follicular lymphoma. Manufacturing succeeded in 99% and the median time from apheresis to delivery was 17 days. The objective response rate was 82% with complete response in 54%; at a median follow-up of 15.4 months 42% remained in response (40% in complete response), and 18-month overall survival was 52%. Grade 3 or higher cytokine release syndrome occurred in 13% and neurological events in 28%. Five-year follow-up showed overall survival of about 43% with a plateau, consistent with cure in a substantial minority. It supported FDA approval in October 2017.

## Fields

- Kind: Key paper
- Last checked: 2026-09-08
- Journal: New England Journal of Medicine
- Year: 2017
- DOI: 10.1056/NEJMoa1707447
- Authors: Neelapu SS, Locke FL, Bartlett NL, et al.
- Findings: 111 enrolled, 101 treated patients with chemotherapy-refractory large B-cell lymphoma; 22 centres.; Manufacturing success 99%; median 17 days from apheresis to delivery; no bridging chemotherapy allowed.; Objective response 82%; complete response 54%; 42% still responding at 15.4 months median follow-up.; 18-month overall survival 52%; 5-year overall survival about 43% with a plateau.; Grade 3 or higher CRS 13%; grade 3 or higher neurological events 28%; 3 deaths during treatment.
- What it means: ZUMA-1 showed that CAR-T can cure a meaningful fraction of adults whose aggressive lymphoma no longer responds to chemotherapy, a group with a historical median survival of about six months (SCHOLAR-1). Axi-cel became the first CAR-T approved for lymphoma and later the first to beat standard second-line therapy in ZUMA-7. The comparatively high neurotoxicity of CD28-costimulated products was also first characterised here.
- Caveats: Single-arm with a historical comparator (SCHOLAR-1).; No bridging therapy was allowed, selecting patients with slower-growing disease.; Roughly 60% of patients eventually relapsed or died; long-term survivors are a minority.; Toxicity required intensive-care-capable centres; real-world use has since expanded with prophylactic steroids.

## Sources

- Full text (DOI): https://doi.org/10.1056/NEJMoa1707447
- ClinicalTrials.gov NCT02348216: https://clinicaltrials.gov/study/NCT02348216

## Connected records

- key papers: [JULIET: tisagenlecleucel for adults with relapsed or refractory diffuse large B-cell lymphoma](https://onco.cc/key-papers/paper-juliet-tisagenlecleucel-dlbcl-nejm-2019/), [Lisocabtagene maraleucel for patients with relapsed or refractory large B-cell lymphomas (TRANSCEND NHL 001): a multicentre seamless design study](https://onco.cc/key-papers/paper-transcend-nhl-001-liso-cel-lancet-2020/), [ZUMA-2: brexu-cel CAR-T for mantle cell lymphoma that has failed BTK inhibitors](https://onco.cc/key-papers/paper-zuma-2-brexu-cel-mantle-cell-nejm-2020/), [ZUMA-7: axi-cel CAR-T instead of salvage chemotherapy and transplant for large B-cell lymphoma that relapses early](https://onco.cc/key-papers/paper-zuma-7-axi-cel-second-line-nejm-2022/)
- roadmaps: [Lymphoma roadmap: from a jaw tumour in Uganda and the first human cancer virus to gene-expression subtypes, PET-adapted chemotherapy, CAR-T cells, bispecific antibodies and the genetics-directed trials now recruiting](https://onco.cc/roadmaps/lymphoma-roadmap/)
- cancers: [Diffuse large B-cell lymphoma](https://onco.cc/cancers/dlbcl/), [Follicular lymphoma](https://onco.cc/cancers/follicular-lymphoma/)
- technologies: [CAR-T cell therapy](https://onco.cc/technologies/car-t/)
- targets: [CD19](https://onco.cc/targets/cd19/)
- drugs: [Axicabtagene ciloleucel](https://onco.cc/drugs/axicabtagene-ciloleucel/)
- companies: [Gilead Sciences (incl. Kite)](https://onco.cc/companies/gilead/)
- institutions: [MD Anderson Cancer Center](https://onco.cc/institutions/md-anderson/), [Moffitt Cancer Center](https://onco.cc/institutions/moffitt/)
- 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: [ZUMA-7](https://onco.cc/trials/zuma-7/)
- people: [Frederick L. Locke](https://onco.cc/people/frederick-locke/), [Michel Sadelain](https://onco.cc/people/michel-sadelain/), [Sattva S. Neelapu](https://onco.cc/people/sattva-neelapu/)
- 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: [New England Journal of Medicine](https://onco.cc/journals/nejm/)
- ideas: [Head-to-head bispecific vs CAR-T in second-line LBCL](https://onco.cc/ideas/idea-bispecific-vs-car-t-second-line/)

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