# Use of Circulating MicroRNAs for Early Detection and Risk Assessment for Pancreatic Cancer

> **NIH NIH R01** · VANDERBILT UNIVERSITY MEDICAL CENTER · 2020 · $638,665

## Abstract

Detecting pancreatic cancer at an early, asymptomatic stage is a top priority for reducing incidence and
mortality of this most fatal disease. MicroRNAs (miRNAs) regulate gene expression and play a critical role in
carcinogenesis and cancer progression. In 2014, two blood miRNA-based diagnostic indices capable of
distinguishing pancreatic cancer patients from healthy controls and chronic pancreatitis patients (AUC: 0.83 to
0.75) were reported. The utility of these indices for pancreatic cancer early detection, however, has not been
evaluated. Recently, we completed a pilot study in which we measured 800 miRNAs in pre-diagnostic plasma
samples from 185 pancreatic cancer cases and individually-matched controls selected from the Prostate, Lung,
Colorectal and Ovary Cancer Screening Trial (PLCO). We applied the algorithms of the two reported diagnostic
indices and found that one index is reasonably effective in discriminating pancreatic cancer patients from
controls within 2 years (AUC= 0.73) but not from 2- <5 years (AUC=0.68) before cancer diagnosis. The AUC
increased to 0.83 for both <2 years and 2- <5 years prior to cancer diagnosis when 4 miRNAs selected
from our own pilot study were applied as the classifier; and for <2 years, further increased to 0.92 when
CA19-9 and known risk factors were added to the classifier. To validate these highly promising findings and to
evaluate the time window during which the discriminative/predictive miRNAs are most informative for cancer
early detection and/or risk assessment, we propose a comprehensive and confirmatory study using additional
PLCO resources as well as resources from four prospective cohort studies: the Southern Community Cohort
Study (SCCS), the Multiethnic Cohort Study (MEC), the Shanghai Women's Health Study (SWHS), and the
Shanghai Men's Health Study (SMHS). Specific aims for the study are: (1) To evaluate all miRNAs showing a
significant association with pancreatic cancer risk in our pilot study and from publications in an independent set
of 341 case-control pairs using plasma collected within 5 years prior to cancer diagnosis. (2) To evaluate the
association of miRNAs replicated in Aim 1 in an independent set of 924 case-control pairs with plasma
samples collected >5 years prior to cancer diagnosis by time windows (e.g., 5-9 years and 10+ years). (3) To
develop indices for early detection (based on miRNAs showing a stronger association in the time window
closer to diagnosis) and risk assessment (based on miRNAs showing a long-term association, e.g., 5+ years
prior to cancer diagnosis) using the PLCO data and validate them using samples from the SCCS, SWHS,
SMHS and MEC. (4) To assess the added value of monitoring secular changes in levels of replicated miRNAs
for cancer early detection using repeated pre-diagnosis plasma samples from the PLCO cases and controls.
Performance of miRNA-based indices will be compared to those based on CA19-9 and TIMP1 and known risk
factors. Built upon strong ...

## Key facts

- **NIH application ID:** 9842619
- **Project number:** 5R01CA227133-02
- **Recipient organization:** VANDERBILT UNIVERSITY MEDICAL CENTER
- **Principal Investigator:** VERONICA WENDY SETIAWAN
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $638,665
- **Award type:** 5
- **Project period:** 2019-01-01 → 2023-12-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9842619

## Citation

> US National Institutes of Health, RePORTER application 9842619, Use of Circulating MicroRNAs for Early Detection and Risk Assessment for Pancreatic Cancer (5R01CA227133-02). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/9842619. Licensed CC0.

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