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Immunotherapy / 免疫治疗Melanoma Combination Immunotherapy & BRAF-Targeted Therapy

Paolo A. Ascierto

保罗·阿西耶托

MD

🏢Istituto Nazionale Tumori IRCCS "Fondazione G. Pascale"(G. Pascale基金会国家肿瘤研究所)🌐Italy

Director, Unit of Melanoma, Cancer Immunotherapy and Innovative Therapy; Vice-Director of Scientific Directorate黑色素瘤、癌症免疫治疗和创新治疗部门主任

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Key Papers
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Awards
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Key Contributions

👥Biography 个人简介

Paolo A. Ascierto, MD is Director of the Unit of Melanoma, Cancer Immunotherapy and Innovative Therapy at the Istituto Nazionale Tumori (INT Pascale) in Naples, Italy, and one of Europe's most prominent melanoma oncologists. He has been a central investigator or steering committee member for the pivotal CheckMate combination immunotherapy trials in melanoma, including CheckMate 067—the landmark phase III trial establishing nivolumab plus ipilimumab as superior to either agent alone in first-line advanced melanoma, achieving unprecedented 5-year overall survival rates of nearly 52%. Dr. Ascierto has also led major investigations into IDO inhibition, tumor vaccines, and novel melanoma immunotherapy combinations, and has been instrumental in the development of cobimetinib and binimetinib as MEK inhibitor partners to BRAF inhibitors. He founded the Society for Melanoma Research and has championed global collaboration in melanoma clinical research.

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🧪Research Fields 研究领域

Nivolumab + Ipilimumab Combination纳武利尤单抗+伊匹木单抗联合治疗
CheckMate Melanoma TrialsCheckMate黑色素瘤试验
BRAF + MEK Inhibitor CombinationsBRAF+MEK抑制剂联合
Tumor Immunology肿瘤免疫学
Cancer Vaccines癌症疫苗

🎓Key Contributions 主要贡献

CheckMate 067: Nivolumab + Ipilimumab Combination Phase III Trial

Served as a principal investigator and steering committee lead for CheckMate 067, demonstrating that nivolumab plus ipilimumab significantly improved progression-free survival (11.5 months) and overall survival compared with ipilimumab alone in treatment-naïve advanced melanoma, with 5-year OS of 52% for the combination—among the highest 5-year survival rates ever reported in metastatic melanoma.

Binimetinib Development (MEK Inhibitor) for NRAS-Mutant and BRAF-Mutant Melanoma

Led clinical development of binimetinib (MEK162), including the MEKTOPO trial and MEKTOPO2 combination studies, demonstrating activity in NRAS-mutant melanoma—a population previously lacking targeted therapy—and contributing to the approval of binimetinib plus encorafenib (BRAF inhibitor) for BRAF V600-mutant melanoma.

IDO Pathway Inhibition and Combination Immunotherapy Research

Led major trials investigating IDO1 inhibitors (epacadostat) in combination with PD-1 blockade in advanced melanoma, contributing critical evidence on the immunosuppressive role of IDO in tumor microenvironment despite the ultimate trial outcomes, and advancing understanding of tryptophan catabolism in immune evasion.

Society for Melanoma Research and Global Collaboration

Founding president of the Society for Melanoma Research (SMR), established the annual SMR Congress as a leading global melanoma forum, and led numerous international melanoma research collaborative initiatives across European and global cancer centers that have accelerated translational-to-clinical pipelines.

Representative Works 代表性著作

[1]

Nivolumab plus Ipilimumab versus Ipilimumab in Untreated Melanoma (CheckMate 067)

New England Journal of Medicine (2015)

Landmark phase III trial demonstrating significantly improved PFS for nivolumab monotherapy and nivolumab plus ipilimumab versus ipilimumab alone, with combination arm achieving 5-year OS of 52%.

[2]

Five-Year Survival Outcomes with Nivolumab plus Ipilimumab versus Ipilimumab in Advanced Melanoma

Journal of Clinical Oncology (2019)

Long-term follow-up of CheckMate 067 demonstrating 5-year OS of 52% for nivolumab plus ipilimumab, establishing unprecedented durable survival in metastatic melanoma.

[3]

Binimetinib versus Dacarbazine in Patients with Advanced NRAS-Mutant Melanoma (NEMO)

The Lancet Oncology (2017)

NEMO phase III trial demonstrating that binimetinib improved PFS versus dacarbazine in NRAS-mutant melanoma, providing the first meaningful targeted therapy data for this patient subset.

[4]

Epacadostat plus Pembrolizumab versus Placebo plus Pembrolizumab in Patients with Unresectable or Metastatic Melanoma (ECHO-301/KEYNOTE-252)

The Lancet Oncology (2019)

Phase III trial demonstrating that IDO1 inhibition with epacadostat did not improve outcomes when added to pembrolizumab in melanoma, but providing fundamental insights into IDO pathway biology in immune-inflamed tumors.

🏆Awards & Recognition 奖项与荣誉

🏆Society for Melanoma Research Award for Scientific Excellence
🏆Italian Association of Medical Oncology (AIOM) Award for Melanoma Research
🏆ESMO Award for Outstanding Contribution to Oncology
🏆Melanoma Research Alliance Distinguished Investigator Award

📄Data Sources 数据来源

Last updated: 2026-04-06 | All information from publicly available academic sources

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