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.

Medina T, Chesney JA, Kluger HM, Hamid O, Whitman ED, Cusnir M, Thomas SS, Wermke M, Domingo-Musibay E, Phan GQ, Kirkwood JM, Larkin J, Weber J, Graf Finckenstein F, Chou J, Gastman B, Wu X, Fiaz R, Sarnaik AA, for the C-144-01 Investigators , Curti B, Kim K, Daniels G, Wilson M, Lee S, Puzanov I, Harker-Murray A, Logan T, Simon JC, Thomas I, Schuler-Thurner B, Moritz R, Hassel J, Grigoliet GU, Arance A, Rubio B, Rodriguez J, Berrocal A, de Sanmamed M, Arkenau HT, Evans T, Corrie P, Dalle S, Bedane C, Olah J, Orcurto A (2025)

Publication Type: Journal article

Publication year: 2025

Journal

Book Volume: 43

Pages Range: 3565-3572

Journal Issue: 33

DOI: 10.1200/JCO-25-00765

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.

Involved external institutions

James Graham Brown Cancer Center United States (USA) (US) Yale University United States (USA) (US) Cedars-Sinai Medical Center United States (USA) (US) Atlantic Health United States (USA) (US) Mount Sinai Medical Center United States (USA) (US) AdventHealth United States (USA) (US) Technische Universität Dresden Germany (DE) Allina Health United States (USA) (US) University of Connecticut Health Center (UConn Health) United States (USA) (US) UPMC Hillman Cancer Center United States (USA) (US) Royal Marsden Hospital / The Royal Marsden NHS Foundation Trust United Kingdom (GB) New York University (NYU) United States (USA) (US) H. Lee Moffitt Cancer Center & Research Institute United States (USA) (US) University of Colorado Cancer Center United States (USA) (US)

How to cite

APA:

Medina, T., Chesney, J.A., Kluger, H.M., Hamid, O., Whitman, E.D., Cusnir, M.,... Orcurto, A. (2025). 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. Journal of Clinical Oncology, 43(33), 3565-3572. https://doi.org/10.1200/JCO-25-00765

MLA:

Medina, Theresa, et al. "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." Journal of Clinical Oncology 43.33 (2025): 3565-3572.

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