Long-Term Efficacy and Safety of Lifileucel Tumor-Infiltrating Lymphocyte Cell Therapy in Patients With Advanced Melanoma: A 5-Year Analysis of the C-144-01 Study

Patients with advanced melanoma resistant to immune checkpoint or BRAF/MEK inhibitors have treatment options with relatively low efficacy. Lifileucel, a one-time autologous tumor-infiltrating lymphocyte cell therapy, was approved in the United States on the basis of the pivotal C-144-01 study. A 5-year follow-up of the C-144-01 trial assessed the long-term efficacy and safety of lifileucel. At the cutoff date (November 20, 2024), the objective response rate was 31.4% (complete response [CR], 5.9%; partial response [PR], 25.5%). Overall, 79.3% of patients had tumor burden reduction; 16 had deepened responses with four converting from PR to CR > 1 year after lifileucel infusion; 31.3% of responders completed the 5-year assessment with ongoing responses. The median duration of response was 36.5 months. Responders (n = 48) had lower tumor burden and fewer liver or brain metastases than the overall population. The median overall survival (OS) was 13.9 months, with a 5-year OS of 19.7%. Adverse events were consistent with nonmyeloablative lymphodepletion and interleukin-2 safety profiles and declined rapidly within 2 weeks after lifileucel infusion. Most grade 3/4 cytopenias resolved to grade ≤2 by day 30. This 5-year analysis demonstrated long-term benefit and meaningful OS with one-time lifileucel therapy, with no additional long-term safety concerns.

Authors

Institutions

Publication Details

Journal
Journal of Clinical Oncology
Published
2025-06-02
DOI
https://doi.org/10.1200/jco-25-00765
Citations
14
Primary Topic
CAR-T cell therapy research
Type
article
Field-Weighted Citation Impact
14.64
Controls
|||
ALL TIME
JAN
FEB
MAR
APR
MAY
JUN
JUL
AUG
SEP
OCT
article

Long-Term Efficacy and Safety of Lifileucel Tumor-Infiltrating Lymphocyte Cell Therapy in Patients With Advanced Melanoma: A 5-Year Analysis of the C-144-01 Study

Harriet M. Kluger, Eric D. Whitman, John M. Kirkwood, Ana Arance et al.
14 citations
Journal of Clinical Oncology
CAR-T cell therapy research
14.64
article

Long-Term Efficacy and Safety of Lifileucel Tumor-Infiltrating Lymphocyte Cell Therapy in Patients With Advanced Melanoma: A 5-Year Analysis of the C-144-01 Study

Harriet M. Kluger, Eric D. Whitman, John M. Kirkwood, Ana Arance, Rana Fiaz, Jeffrey Chou, Ioannis Thomas, Miguel F. Sanmamed, Amy Harker-Murray, Omid Hamid, Brendan D. Curti, Theresa Michelle Medina, Gregory A. Daniels, Amod A. Sarnaik, Martin Wermke, Giao Q. Phan, Jeffrey S. Weber, Angela Orcurto, Igor Puzanov, Rose K. C. Moritz, Jan C. Simon, Mike Cusnir, Thomas Evans, Pippa Corrie, Sajeve Thomas, Jessica Cecile Hassel, Alfonso Berrocal, Evidio Domingo‐Musibay, Stéphane Dalle, B. Rubio, James Larkin, Jason Chesney, Christophe Bédane, Brian Gastman, Beatrice Schuler-Thurner, Xiao Wu, Melissa Wilson, Sylvia Lee, Kevin Kim, Friedrich Graf Finckenstein, Theodore F. Logan, Juan C. Rodríguez, Hendrik-Tobias Arkenau, Judit Oláh, Gotz Ulrich Grigoliet
article en
14 citations

Abstract

Patients with advanced melanoma resistant to immune checkpoint or BRAF/MEK inhibitors have treatment options with relatively low efficacy. Lifileucel, a one-time autologous tumor-infiltrating lymphocyte cell therapy, was approved in the United States on the basis of the pivotal C-144-01 study. A 5-year follow-up of the C-144-01 trial assessed the long-term efficacy and safety of lifileucel. At the cutoff date (November 20, 2024), the objective response rate was 31.4% (complete response [CR], 5.9%; partial response [PR], 25.5%). Overall, 79.3% of patients had tumor burden reduction; 16 had deepened responses with four converting from PR to CR > 1 year after lifileucel infusion; 31.3% of responders completed the 5-year assessment with ongoing responses. The median duration of response was 36.5 months. Responders (n = 48) had lower tumor burden and fewer liver or brain metastases than the overall population. The median overall survival (OS) was 13.9 months, with a 5-year OS of 19.7%. Adverse events were consistent with nonmyeloablative lymphodepletion and interleukin-2 safety profiles and declined rapidly within 2 weeks after lifileucel infusion. Most grade 3/4 cytopenias resolved to grade ≤2 by day 30. This 5-year analysis demonstrated long-term benefit and meaningful OS with one-time lifileucel therapy, with no additional long-term safety concerns.

Journal of Clinical OncologyVol. 43(33)
Allina Health (US), AdventHealth Orlando (US), Yale Cancer Center (US), Moffitt Cancer Center (US), NYU Langone Health (US), Mount Sinai Medical Center (US), Angeles Clinic and Research Institute (US), Royal Marsden Hospital (GB), UPMC Hillman Cancer Center (US), University of Colorado Cancer Center (US), James Graham Brown Foundation (US), Atara Biotherapeutics (United States) (US), Atlantic Health System (US), UConn Health (US), Technische Universität Dresden (DE), University of Colorado Denver (US)
Good health and well-being
Openalex Percentile: Top 1%
CAR-T cell therapy research
14.64
DOISourceOpenAlex
AI Navigator

Ask Laika to Summarize, Analyze, and Connect papers live on the map.

Summarize Papers & Methodologies

Extract key findings, datasets, and comparative methods across publications.

Benchmark Rankings & Visual Analytics

Rank top research institutions, authors, funders, topics, and journals by Field-Weighted Citation Impact (FWCI) and paper volume with instant charts.

Connect Distant Disciplines

Bridge topological clusters on the map to find hidden collaborative intersections.