Background: Patients with pancreatic ductal adenocarcinoma (PDAC) exhibit a limited response to standard-of-care chemotherapy. The cancer secretome may offer novel therapeutic targets to enhance current treatments.
Objective: Through multi-modal profiling of PDAC cells, we aimed to identify secreted proteins contributing to PDAC progression and metastasis, to reveal new candidates for co-targeting with standard-of-care chemotherapy.
Design: RNA sequencing (RNAseq) and mass spectrometry (MS)-based proteomics assessed the transcriptome, secretome and exosome profile of PDAC cells from the highly-metastatic KPC and the poorly-metastatic KPflC genetically engineered mouse models (GEMMs). Proprotein convertase subtilisin/kexin type 9 (PCSK9) was identified as a potential co-target and was subsequently validated in PDAC patient cohorts. PCSK9 inhibition was evaluated in combination with standard-of-care gemcitabine/nab-paclitaxel (GnP) chemotherapy using in vitro and in vivo models of PDAC. Therapy response was visualised via multiphoton imaging of the fluorescent ubiquitination-based cell cycle indicator (FUCCI) reporter.
Results: PCSK9 expression and secretion was enhanced in highly-metastatic PDAC cells. High PCSK9 expression was associated with increased histopathological tumour grade and worse survival in PDAC patients. PCSK9 inhibition impeded cancer cell invasion, reduced tumour growth in vivo, and further sensitised cells to GnP. RNAseq revealed a dysregulation of cell cycle-associated genes, confirmed by FUCCI multiphoton microscopy. In vitro experiments and in vivo models of metastasis revealed that PCSK9 inhibition combined with GnP significantly reduced cancer cell survival and liver metastasis.
Conclusion: This study highlights an anti-cancer role for PCSK9 inhibition in combination with GnP chemotherapy for pancreatic cancer.