# ELIANA: the global trial that made tisagenlecleucel the first approved CAR-T therapy for children and young adults with relapsed ALL

Source: https://onco.cc/key-papers/paper-eliana-tisagenlecleucel-nejm-2018/  
OnCo record `paper-eliana-tisagenlecleucel-nejm-2018` (Key paper). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Across 25 centres, 81% of children and young adults with relapsed or refractory ALL went into remission after a single tisagenlecleucel infusion, and half were still event-free a year later.

## Summary

ELIANA was a single-arm, multicentre phase 2 trial of tisagenlecleucel in patients aged 3-21 with CD19-positive relapsed or refractory B-cell ALL. Of 92 enrolled, 75 received an infusion; the rest could not because of manufacturing failure, death or adverse events. The overall remission rate within three months was 81%, all MRD-negative; event-free survival was 73% at six months and 50% at 12 months, with overall survival 90% and 76%. Cytokine release syndrome occurred in 77% (grade 3-4 in about 46%) and neurological events in 40%. The trial supported FDA approval in August 2017, the first gene therapy and first CAR-T approved in the United States, and demonstrated that a centrally manufactured autologous cell product could be delivered across continents.

## Fields

- Kind: Key paper
- Last checked: 2026-09-08
- Journal: New England Journal of Medicine
- Year: 2018
- DOI: 10.1056/NEJMoa1709866
- Authors: Maude SL, Laetsch TW, Buechner J, et al.
- Findings: 92 enrolled, 75 infused; 25 centres in 11 countries; median age 11.; Overall remission rate within 3 months 81%; all remissions MRD-negative.; Event-free survival 73% at 6 months and 50% at 12 months; overall survival 90% and 76%.; CRS 77% (grade 3-4 in about 46%; 48% needed tocilizumab; 47% intensive care); neurological events 40%.; Manufacturing failed in 7 of 92 and 17 enrolled patients were never infused, highlighting the vein-to-vein problem.
- What it means: ELIANA turned CAR-T from a single-centre experiment into a licensed product and created the regulatory and logistical template every later cell therapy has followed. For children with refractory leukaemia it offers a chance of durable remission without transplant. The trial also exposed the gaps: manufacturing failures, patients dying while waiting, and roughly half relapsing within a few years.
- Caveats: Single-arm; no randomised comparator and outcomes reported on infused rather than enrolled patients in most analyses.; Severe CRS rates were high by later standards, before prophylactic strategies.; Long-term relapse, often CD19-negative, affects about half; some patients still proceed to transplant.; List price around 475,000 US dollars at launch raised affordability and access questions.

## Sources

- Full text (DOI): https://doi.org/10.1056/NEJMoa1709866
- ClinicalTrials.gov NCT02435849: https://clinicaltrials.gov/study/NCT02435849

## Connected records

- key papers: [Maude 2014: CD19 CAR-T cells produce complete remission in 27 of 30 children and adults with relapsed ALL](https://onco.cc/key-papers/paper-maude-ctl019-all-nejm-2014/), [UCART19: the first gene-edited, donor-derived CAR-T cells in children and adults with relapsed B-cell ALL](https://onco.cc/key-papers/paper-ucart19-allogeneic-car-t-lancet-2020/)
- technologies: [Apheresis and starting-material collection](https://onco.cc/technologies/apheresis-starting-material/), [CAR-T cell therapy](https://onco.cc/technologies/car-t/)
- cancers: [Acute lymphoblastic leukaemia](https://onco.cc/cancers/all-leukemia/), [Relapsed and refractory acute lymphoblastic leukaemia in children](https://onco.cc/cancers/all-paediatric-relapsed/)
- targets: [CD19](https://onco.cc/targets/cd19/)
- drugs: [Obecabtagene autoleucel](https://onco.cc/drugs/obecabtagene-autoleucel/), [Tisagenlecleucel](https://onco.cc/drugs/tisagenlecleucel/)
- companies: [Novartis](https://onco.cc/companies/novartis/)
- institutions: [Abramson Cancer Center, University of Pennsylvania](https://onco.cc/institutions/penn-abramson/)
- terms: [Cytokine release syndrome (CRS)](https://onco.cc/terms/crs/), [Event-free / disease-free survival (EFS, DFS, iDFS, RFS)](https://onco.cc/terms/efs/), [ICANS (neurotoxicity)](https://onco.cc/terms/icans/)
- trials: [ELIANA](https://onco.cc/trials/eliana/)
- people: [Bruce L. Levine](https://onco.cc/people/bruce-levine/), [Carl H. June](https://onco.cc/people/carl-june/), [Stephan A. Grupp](https://onco.cc/people/stephan-grupp/)
- bottlenecks: [Manufacturing cost and time for living and radioactive medicines](https://onco.cc/bottlenecks/b-manufacturing-cell-therapy/), [Most of the world has almost no cancer care](https://onco.cc/bottlenecks/b-global-access/), [Prices and value](https://onco.cc/bottlenecks/b-drug-pricing/)
- journals: [New England Journal of Medicine](https://onco.cc/journals/nejm/)

---
JSON: https://onco.cc/api/v1/entities/paper-eliana-tisagenlecleucel-nejm-2018.json