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Dung T. Le

First author of the studies that showed mismatch-repair-deficient tumours respond to PD-1 blockade regardless of origin.

Led the KEYNOTE-016 study and its extension establishing pembrolizumab's tissue-agnostic activity in dMMR tumours; also works on GVAX and pancreatic cancer vaccines.

Role
Professor of Oncology; Co-Director, GI Cancer Immunotherapy
Specialisms
MSI-high cancersPancreatic cancer immunotherapyCancer vaccines

Papers

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TitleJournalYear
PD-1 blockade in tumors with mismatch-repair deficiencyNew England Journal of Medicine2015

Key papers

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rctNew England Journal of Medicine 2020changed practice
KEYNOTE-177: pembrolizumab instead of chemotherapy as first treatment for mismatch-repair-deficient metastatic colorectal cancer

Every colorectal cancer should be tested for mismatch repair deficiency, because patients whose metastatic tumour is dMMR should receive pembrolizumab rather than chemotherapy as first treatment, gaining a much better chance of durable remission with fewer side effects. About a third of dMMR tumours do not respond initially, so early scans are essential and chemotherapy remains available. The trial does not apply to the 95% of colorectal cancers that are mismatch-repair proficient.

translationalScience 2017changed practice
Le 2017: mismatch-repair deficiency predicts response to PD-1 blockade across twelve tumour types, leading to the first tissue-agnostic drug approval

This paper established a new regulatory paradigm: a drug approved for a molecular feature regardless of organ. It made MSI/MMR testing standard across advanced cancers and remains the clearest example of a biomarker that works across histologies. It also anchored the idea that mutation load, via neoantigens, is what makes tumours visible to T cells.

translationalNew England Journal of Medicine 2015changed practice
Le 2015: PD-1 blockade works in tumours with mismatch-repair deficiency, whatever the organ

This small trial explained why colorectal cancer had seemed immune-resistant (most is MMR-proficient) and established the principle that a genomic feature, not the tissue of origin, can predict immunotherapy response. It led directly to the 2017 tissue-agnostic approval of pembrolizumab and to routine MMR/MSI testing of many cancers. Every patient with advanced dMMR cancer should now be considered for checkpoint blockade.

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