T-cell receptor therapy (TCR-T) is a cell-based immunotherapy that targets human leukocyte antigen (HLA)-presented neoantigens using naturally occurring TCRs, enabling targeted immune-activation at very low-antigen density [1]. This ability to target intracellular-antigens and activate immune responses at low-antigen levels gives TCR-T an advantage over chimeric antigen receptor therapy (CAR-T), especially in treating solid-tumours and cancers with limited surface antigens [2]. TCR-T requires identification of cancer-specific HLA-presented tumour neoantigens (TNA) and corresponding high-affinity TCRs against these HLA-presented TNAs. Our team has developed an integrated multi-omics TNA discovery platform, combining immunopeptidomics, genomics, and immunologic techniques. We successfully identified an immunogenic mutant HLA-presented TNA in aggressive immunologically "cold" ovarian cancer with low tumour mutational burden: neuropeptide W E100Q (NPW_E100Q), restricted to HLA-A*33:03.
Using single-cell RNA sequencing (scRNAseq), we discovered three high-affinity TCRs against NPW_E100Q in context of HLA-A*33:03. First, we optimised the antigen presenting cell (dendritic cell; DC) and CD8+ T-cell co-culture conditions (14-day culture with IL-7/IL-15/IL-21) to selectively expand NPW_E100Q-specific CD8+ T-cells, utilising HLA-matched healthy donor blood. We demonstrated that CD8+ T-cells expanded in vitro in the presence of DC pulsed with NPW_E100Q peptide showed increased NPW_E100Q-specific CD8+ T-cell proliferation, identified by the presence of NPW_E100Q-specific dextramer staining compared to wild-type NPW dextramer. The proliferated CD8+ T-cells were fluorescence-activated cell sorted. Subsequent scRNAseq revealed 1,023 paired α/β-TCR clonotypes from the CD8+ T-cells co-cultured with NPW_E100Q peptide-pulsed DCs; showing high specificity with <28 TCRs shared between NPW_E100Q and wild-type NPW stimulations. High-affinity TCRs were identified as the α/β-TCR pairs of the most abundant clonotypes. TCRs with cross-reactivity against wild-type NPW and expression of TCR gamma chains were excluded. Lastly, we confirmed the specificity of T-cell responses by AIM (Antigen Identification by MHC) analysis. We are now in the process of engineering precision TCR-T onto donor CD8+ T-cells for in vivo cytocidal testing.
In conclusion, we confirmed our ability to identify immunogenic HLA-presented TNAs and their corresponding high-affinity TCRs. We plan to expand this study into a cohort of well-curated pancreatic biospecimens with matched genomic and clinical data. This work has the potential to revolutionise the solid tumour treatment landscape with precision cell-based immunotherapy.