F. Stephen Hodi
First author of the 2010 ipilimumab trial that proved immunotherapy could extend survival in melanoma.
Led the phase 3 ipilimumab study that opened the checkpoint-inhibitor era, and later the CheckMate 067 nivolumab-ipilimumab long-term follow-up; directs Dana-Farber's immuno-oncology centre.
| Title | Journal | Year |
|---|---|---|
| Improved survival with ipilimumab in patients with metastatic melanoma | New England Journal of Medicine | 2010 |
| Long-term survival with nivolumab plus ipilimumab or nivolumab alone in advanced melanoma (CheckMate 067) | New England Journal of Medicine | 2025 |
Before 2011 median survival in metastatic melanoma was under a year; this trial shows that roughly half of patients treated with combination checkpoint blockade are now long-term survivors, effectively cured. It anchors first-line treatment of advanced melanoma and sets the benchmark for every new regimen, including nivolumab-relatlimab. The combination's toxicity means nivolumab alone or newer doublets remain reasonable for some patients.
For patients with advanced melanoma, immunotherapy offers a realistic chance of long-term survival and probably cure, and the ten-year data show that patients who are alive and progression-free at three years rarely die of melanoma afterwards. Nivolumab plus ipilimumab gives the best long-term results but at a high price in serious side effects; nivolumab alone or nivolumab plus relatlimab are alternatives for patients at lower risk or with autoimmune concerns. The trial is also a caution about surrogate endpoints: the survival plateau took years to become visible.
This study is why PD-1 inhibitors were developed across cancers rather than in melanoma alone: unexpected activity in lung cancer, historically thought immune-resistant, changed drug development priorities industry-wide. It also introduced PD-L1 immunohistochemistry as a candidate biomarker and pneumonitis as a signature toxicity. Within five years PD-1 blockade was approved in more than ten cancers.
This paper launched the immunotherapy era. It was the first randomised evidence that taking a brake off the immune system could extend life in a solid cancer, and it introduced clinicians to immune-related adverse events and to responses that arrive late or after apparent progression. Ipilimumab alone has since been superseded by PD-1 antibodies and combinations, but every checkpoint programme traces back to this result.