Background
Biomarkers of therapeutic response and prognosis are needed to assist in the sequencing of treatments for metastatic castration-resistant prostate cancer (CRPC). MicroRNAs are regulators of gene expression, and are shown to be involved in tumour progression. Previously in a Phase 1 discovery study of 97 patients with CRPC, we identified 14 circulating microRNAs that were associated with response to docetaxel chemotherapy or overall survival. We performed a Phase 2 validation study to verify these findings.
Methods
Using real-time PCR and Taqman assays, the levels of the 14 microRNAs were measured in plasma collected before and after the first cycle of docetaxel from a Phase 2 cohort of 89 patients with CRPC.
Results
None of the microRNAs were associated with docetaxel response in the Phase 2 cohort. Higher baseline levels of six microRNAs, predominantly of the miR-200 family, were confirmed to be associated with shorter overall survival. A microRNA signature comprising these six microRNAs predicted high risk patients in the Phase 2 cohort with a hazard ratio of 4.12 (95% CI 2.20-7.70, P = 0.000001). The signature was an independent predictor (P = 0.001) in multivariable analysis with clinicopathological factors (hemoglobin P = 0.06; alkaline phosphatase P = 0.85; serum prostate serum antigen, P = 0.60) in the Phase 2 cohort.
Conclusions
The association of the six microRNAs with prognosis suggests their involvement in CRPC pathogenesis. Further research is required to determine if these microRNAs could be targeted with microRNA-based therapeutics to improve patient outcome.