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Neoantigen

A protein fragment created by a tumour mutation that the immune system has never seen before, so it can attack it without harming normal cells.

Arise from missense mutations, frameshifts, fusions, and splice variants; must be processed and presented on the patient's HLA. Predicted computationally (binding affinity, expression, clonality). The targets of personalised mRNA vaccines and neoantigen-specific TCR-T; higher TMB means more candidates.

Immunology: what this kind of term is about · animated schematic, not to scale
Category
Immunology

Key papers

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rctThe Lancet 2024
KEYNOTE-942: a personalised mRNA cancer vaccine plus pembrolizumab after melanoma surgery

For the first time a randomised trial suggests that a vaccine tailored to an individual's tumour can reduce relapse when combined with immunotherapy, which is a proof of concept for a field that had failed for decades. Nothing changes for patients yet: the trial was small, the confidence interval crossed one, and the phase 3 trial in melanoma (and parallel trials in lung and other cancers) must confirm it. If it does, personalised mRNA vaccines could become a routine adjunct to checkpoint inhibitors after surgery.

translationalNature 2023
Rojas 2023: a personalised mRNA vaccine trained T cells against each patient's pancreatic cancer, and those who responded stayed cancer-free longer

Pancreatic cancer was thought to be immunologically inert because of its low mutation burden; this study showed that with the right vaccine platform its few neoantigens can still be targeted. It is the strongest human evidence so far that personalised cancer vaccines can generate durable, tumour-specific immunity, and it justifies the randomised trials now running in pancreatic cancer and melanoma. Benefit is not yet proven, because responders may simply have had more immunogenic tumours.

translationalClinical Cancer Research 2020
First trial of a vaccine against the shared neoantigens of mismatch-repair-deficient cancers

Because Lynch syndrome tumours make the same abnormal proteins in almost every patient, a single vaccine could in principle be given to carriers before cancer develops. This small trial showed the concept is safe and immunogenic; whether it prevents cancer requires the randomised trials now being planned.

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 2017
TRACERx first 100: tracking how lung cancers evolve, and how chromosomal chaos predicts relapse

Lung cancers keep evolving after they form, and it is ongoing chromosomal instability rather than the number of mutations that best predicts who will relapse. This gives a rationale for targeting the earliest (clonal) drivers and neoantigens and for tracking evolution in blood after surgery.

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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