# C-144-01: lifileucel, tumour-infiltrating lymphocytes grown from a patient's own tumour, in melanoma after checkpoint inhibitors have failed

Source: https://onco.cc/key-papers/paper-c-144-01-lifileucel-melanoma-jco-2021/  
OnCo record `paper-c-144-01-lifileucel-melanoma-jco-2021` (Key paper). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Immune cells harvested from a patient's tumour, expanded in the lab and reinfused shrank melanoma in 36% of patients whose disease had progressed on PD-1 blockade, with responses that mostly lasted.

## Summary

Cohort 2 of the C-144-01 phase 2 trial treated 66 patients with advanced melanoma that had progressed after anti-PD-1 therapy (and BRAF/MEK inhibitors where indicated; median 3.3 prior lines) with a single infusion of lifileucel, autologous tumour-infiltrating lymphocytes expanded centrally over about 22 days, after non-myeloablative lymphodepletion and followed by up to six doses of high-dose interleukin-2. The objective response rate was 36% (2 complete, 22 partial) with disease control in 80%; median duration of response was not reached at 18.7 months of follow-up. Adverse events were largely attributable to lymphodepletion and IL-2 and resolved within two weeks, with two treatment-related deaths. Pooled analysis of 153 patients from cohorts 2 and 4 showed a 31% response rate and supported FDA accelerated approval in February 2024, the first TIL therapy and first cell therapy for a solid tumour.

## Fields

- Kind: Key paper
- Last checked: 2026-09-08
- Journal: Journal of Clinical Oncology
- Year: 2021
- DOI: 10.1200/JCO.21.00612
- Authors: Sarnaik AA, Hamid O, Khushalani NI, et al.
- Findings: 66 patients with anti-PD-1-refractory advanced melanoma; median 3.3 prior therapies; single lifileucel infusion after cyclophosphamide-fludarabine lymphodepletion, then up to 6 doses of high-dose IL-2.; Objective response 36% (3% complete); disease control 80%.; Median duration of response not reached at 18.7 months; responses deepened over time in some patients.; Grade 3-4 adverse events mostly cytopenias, febrile neutropenia and IL-2-related hypotension, resolving within 2 weeks; 2 treatment-related deaths.; Pooled cohorts 2 and 4 (153 patients): objective response 31.4%, supporting accelerated approval in 2024.
- What it means: Lifileucel proved that Steven Rosenberg's decades-old TIL concept could be industrialised into a licensed product and gave patients with checkpoint-refractory melanoma, who otherwise have few options, a chance of durable remission. It is the first cell therapy approved for any solid tumour. The treatment requires surgery to harvest tumour, hospitalisation for lymphodepletion and IL-2, and specialised centres, so its reach is limited.
- Caveats: Single-arm trial; approval rested on response rate rather than survival.; Toxic conditioning and high-dose IL-2 make the regimen unsuitable for frail patients.; Manufacturing takes about three weeks and needs a resectable lesion; not all patients yield a product.; Cost (list price above 500,000 US dollars) and centre requirements restrict access.

## Sources

- PubMed search: https://pubmed.ncbi.nlm.nih.gov/?term=C-144-01%20lifileucel%20tumor-infiltrating%20lymphocyte%20melanoma%20Sarnaik%20JCO%202021
- ClinicalTrials.gov NCT02360579: https://clinicaltrials.gov/study/NCT02360579

## Connected records

- key papers: [Rohaas 2022: the first randomised trial of TIL therapy, against ipilimumab, in advanced melanoma](https://onco.cc/key-papers/paper-rohaas-til-vs-ipilimumab-nejm-2022/), [SPEARHEAD-1: afamitresgene autoleucel, the first engineered T-cell receptor therapy approved for a solid tumour, in synovial sarcoma](https://onco.cc/key-papers/paper-spearhead-1-lancet-2024/)
- cancers: [Advanced melanoma (unresectable stage III and stage IV)](https://onco.cc/cancers/advanced-melanoma/), [Melanoma](https://onco.cc/cancers/melanoma/)
- technologies: [TIL therapy](https://onco.cc/technologies/til-therapy/)
- drugs: [Aldesleukin (high-dose IL-2)](https://onco.cc/drugs/aldesleukin/), [Cyclophosphamide](https://onco.cc/drugs/cyclophosphamide/), [Lifileucel](https://onco.cc/drugs/lifileucel/)
- companies: [Iovance Biotherapeutics](https://onco.cc/companies/iovance/)
- institutions: [Moffitt Cancer Center](https://onco.cc/institutions/moffitt/), [National Cancer Institute (NIH)](https://onco.cc/institutions/nci/)
- terms: [Objective response rate (ORR)](https://onco.cc/terms/orr/), [Tumour-infiltrating lymphocytes (TILs)](https://onco.cc/terms/tils/)
- trials: [C-144-01](https://onco.cc/trials/c-144-01/)
- people: [Amod A. Sarnaik](https://onco.cc/people/amod-sarnaik/), [Steven A. Rosenberg](https://onco.cc/people/steven-rosenberg/)
- bottlenecks: [Manufacturing cost and time for living and radioactive medicines](https://onco.cc/bottlenecks/b-manufacturing-cell-therapy/), [No one can predict who responds to immunotherapy](https://onco.cc/bottlenecks/b-immunotherapy-response/)
- journals: [Journal of Clinical Oncology](https://onco.cc/journals/jco/)

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