Objective/rationale:
Pancreatic ductal adenocarcinoma (PDAC) is a highly lethal malignancy that urgently needs more effective therapies. Stromal cancer-associated fibroblasts (CAFs) play a major role in promoting tumour progression and chemoresistance, by creating a fibrotic tumour microenvironment that physically impedes drug access, and feeds PDAC cells nutrients and pro-tumour signals. Thus, in partnership with Noxopharm Limited we have developed a drug (CRO-67) designed to target both PDAC cells and CAFs. Aims: 1) Investigate the therapeutic potential of CRO-67 in patient-derived PDAC tumour explant model, mimicking the heterogeneity and multicellular architecture of human disease. 2) Assess the effect of CRO-67 on PDAC cell and CAF proliferation, apoptosis, and cell cycle progression to elucidate the mode of action.
Methods:
1) PDAC tumour samples were collected from 7 patients undergoing pancreatic resection. Tumour explants (1 – 2 mm diameter) were cultured on gelatin sponges and treated with CRO-67 (0 – 50 μg/mL) every 3 days and fixed on day 12. Therapeutic response was assessed by immunohistochemistry for markers of tumour cells (cytokeratin), CAFs (α-Smooth Muscle Actin), cell proliferation (Bromodeoxyuridine), and cell death (TUNEL).
2) PDAC cells and patient-derived CAFs were treated with CRO-67 for 24 h before cell proliferation (IncuCYTE S3) and apoptosis (Annexin V/DAPi staining and flow cytometry) analyses, or for 4 h before cell cycle analysis (flow cytometry).
Results:
1) CRO-67 treatment decreased tumour and CAF cell frequency in 5/7 and 6/7 patient PDAC tumour explants, respectively. It also decreased cell proliferation, and increased cell death relative to controls. 2) CRO-67 reduced proliferation of CAFs (IC50 = 346.9 nM) and PDAC cells (IC50 = 251.5 nM), increased apoptosis by 219 ± 6.5% (p<0.0001) and increased the fraction of cells in the G2/M cell cycle phase by 37 ± 3.6% (p<0.0001) relative to controls.
Conclusions:
Our findings demonstrate that CRO-67: 1) reduced PDAC cells and CAFs in human tumour explants; 2) reduced proliferation of PDAC cells and CAFs, increased apoptosis, and disrupted cell cycle progression through G2/M phase. Implication: CRO-67 is an ideal candidate for further preclinical investigation in vivo. We predict that this drug could lead to a more effective therapy for pancreatic cancer, able to directly kill PDAC cells and alleviate physical barriers to drug delivery by targeting CAFs, allowing sensitisation to chemotherapy.