# Axicabtagene ciloleucel

Source: https://onco.cc/drugs/axicabtagene-ciloleucel/  
OnCo record `axicabtagene-ciloleucel` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

A CD19 CAR-T that cures about 40% of patients with large B-cell lymphoma who had failed everything, and beat transplant in second line.

## Summary

Axicabtagene ciloleucel (axi-cel) is an autologous CAR-T: a patient's T cells are engineered with a retroviral vector to express a CD19-binding scFv with a CD28 costimulatory domain and reinfused after cyclophosphamide and fludarabine lymphodepletion. It was approved in 2017 for large B-cell lymphoma after two or more lines (ZUMA-1; 5-year OS about 43%), in 2022 as second-line therapy for early relapse (ZUMA-7, OS benefit), and for follicular lymphoma. Kite/Gilead manufactures it. CD28 costimulation gives fast expansion and more neurotoxicity than 4-1BB products; cytokine release syndrome, prolonged cytopenias and infections are the other main risks. How to choose between CAR-T and CD20xCD3 bispecifics, and how to widen access, remain open. For a newcomer: a one-time living drug that cures a meaningful share of lymphomas with no standard treatment left to try.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-04
- Brand: Yescarta
- Code: axi-cel
- Modality: CAR-T (CD19)
- Mechanism: CD19 scFv, CD28 costimulation, retroviral.
- Approvals: US 2017: Relapsed/refractory large B-cell lymphoma ≥2 lines; US 2022: Second-line LBCL refractory or relapsed within 12 months
- Dosing: Single IV infusion after lymphodepletion; 2 × 10⁶ CAR+ T cells/kg (max 2 × 10⁸); cyclophosphamide 500 mg/m² + fludarabine 30 mg/m² days −5 to −3

## Notes

- Lymphoma biology: half the medicine is the patient's own T cells. Single-cell sequencing of 24 infusion products found three times the frequency of memory-signature CD8 T cells in patients who were in complete response at three months, and an exhaustion signature associated with a poor molecular response in cell-free DNA at day 7; a rare monocyte-like population in the product was associated with high-grade neurotoxicity (Deng 2020). The other failure mode is CD19 target evasion (Plaks 2021, Sotillo 2015).

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Axicabtagene_ciloleucel
- FDA label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Axicabtagene%20ciloleucel
- Deng et al., Nat Med 2020: single-cell features of the axicabtagene ciloleucel infusion product that track efficacy and toxicity in 24 patients: https://doi.org/10.1038/s41591-020-1061-7
- Plaks et al., Blood 2021: CD19 target evasion as a mechanism of relapse after axicabtagene ciloleucel in large B-cell lymphoma: https://doi.org/10.1182/blood.2021010930
- Sotillo et al., Cancer Discov 2015: acquired mutations and alternative splicing of CD19 enable resistance to CART-19: https://doi.org/10.1158/2159-8290.CD-15-1020

## Connected records

- cancers: [Burkitt lymphoma](https://onco.cc/cancers/burkitt-lymphoma/), [Diffuse large B-cell lymphoma](https://onco.cc/cancers/dlbcl/), [Follicular lymphoma](https://onco.cc/cancers/follicular-lymphoma/), [HIV-associated (AIDS-related) lymphomas](https://onco.cc/cancers/hiv-associated-lymphoma/), [Non-Hodgkin lymphoma (all types)](https://onco.cc/cancers/non-hodgkin-lymphoma/), [Post-transplant lymphoproliferative disorder (PTLD)](https://onco.cc/cancers/post-transplant-lymphoproliferative-disorder/), [Primary CNS lymphoma](https://onco.cc/cancers/primary-cns-lymphoma/), [Primary mediastinal (thymic) large B-cell lymphoma](https://onco.cc/cancers/primary-mediastinal-b-cell-lymphoma/)
- technologies: [Autologous CAR-T manufacturing, batch by batch](https://onco.cc/technologies/car-t-manufacturing-process/), [CAR-T cell therapy](https://onco.cc/technologies/car-t/)
- targets: [CD19](https://onco.cc/targets/cd19/), [CD28](https://onco.cc/targets/cd28/)
- companies: [Gilead Sciences (incl. Kite)](https://onco.cc/companies/gilead/), [LYSA (The Lymphoma Study Association)](https://onco.cc/companies/lysa/)
- terms: [Antigen escape: how a lymphoma loses the thing the drug was aimed at](https://onco.cc/terms/lymphoma-bio-antigen-escape/), [Cytokine release syndrome and ICANS: grading and management](https://onco.cc/terms/lymphoma-tx-crs-icans/), [Immunoglobulin replacement after CAR-T, bispecifics and long anti-CD20 treatment](https://onco.cc/terms/lymphoma-tx-immunoglobulin-replacement/), [Lymphoma (tissue type)](https://onco.cc/terms/lymphoma-type/), [POD24: progression of follicular lymphoma within two years, and why it changes the plan](https://onco.cc/terms/lymphoma-tx-pod24/), [The CAR-T pathway in lymphoma: referral, apheresis, bridging and the waiting](https://onco.cc/terms/lymphoma-tx-car-t-pathway/), [What the NHS in England funds for lymphoma, appraisal by appraisal](https://onco.cc/terms/lymphoma-tx-uk-access/)
- trials: [Emapalumab Prevention of CAR-T Cell Associated Toxicities](https://onco.cc/trials/nct06550141/), [Study of Axicabtagene Ciloleucel Versus Standard of Care Therapy in Participants With Relapsed/Refractory Follicular Lymphoma](https://onco.cc/trials/nct05371093/), [ZUMA-1](https://onco.cc/trials/zuma-1/), [ZUMA-5](https://onco.cc/trials/zuma-5/), [ZUMA-7](https://onco.cc/trials/zuma-7/)
- ideas: [Full refund for CAR-T if the patient has not responded at three months](https://onco.cc/ideas/idea-reg-cart-outcomes-refund/), [Head-to-head bispecific vs CAR-T in second-line LBCL](https://onco.cc/ideas/idea-bispecific-vs-car-t-second-line/), [Mandatory public reporting of vein-to-vein time and failure rate per CAR-T product](https://onco.cc/ideas/idea-reg-vein-to-vein-public-benchmark/), [Measure the late effects of the treatments being given now, not the ones given in 1975](https://onco.cc/ideas/lymphoma-ev-late-effects-of-the-treatments-given-now/), [Outcome-based annuity payments for potentially curative one-time therapies](https://onco.cc/ideas/idea-fund-pay-for-cure-annuities/), [Pay-for-cure contracts: instalment payments for curative therapies contingent on durable remission](https://onco.cc/ideas/idea-moon-pay-for-cure-contracts/), [Report the time from apheresis to infusion as a trial endpoint, not a logistics footnote](https://onco.cc/ideas/lymphoma-ev-manufacturing-time-as-a-trial-endpoint/)
- key papers: [Axicabtagene ciloleucel in relapsed or refractory indolent non-Hodgkin lymphoma (ZUMA-5): a single-arm, multicentre, phase 2 trial](https://onco.cc/key-papers/paper-zuma-5-axi-cel-indolent-lymphoma-lancet-oncol-2022/), [ZUMA-1: axicabtagene ciloleucel for refractory large B-cell lymphoma, the first CAR-T approved for lymphoma](https://onco.cc/key-papers/paper-zuma-1-axi-cel-nejm-2017/), [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: [Cell therapy roadmap: CD19 CAR-T → solid tumours → in vivo CAR](https://onco.cc/roadmaps/cell-therapy-roadmap/), [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/)
- people: [Frederick L. Locke](https://onco.cc/people/frederick-locke/), [Jason R. Westin](https://onco.cc/people/jason-westin/), [Sattva S. Neelapu](https://onco.cc/people/sattva-neelapu/)
- biomarkers: [CD19 expression (CD19-positive)](https://onco.cc/biomarkers/cd19-expression/)

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