# MicroRNA Biomarkers for Determining Treatment Response in Colorectal Cancer

> **NIH NIH R01** · BECKMAN RESEARCH INSTITUTE/CITY OF HOPE · 2020 · $405,048

## Abstract

Colorectal cancer (CRC) is a common malignancy, and remains the second leading cause of
cancer-related deaths in the United States. The present guidelines for CRC treatment recommend that
patients with stage III and IV colon cancers (with lymph node or distant metastasis) should be treated
with adjuvant or palliative chemotherapy using cytotoxic drugs such as 5-fluorouracil (5-FU) and
oxaliplatin after surgical resection of the primary cancer.In spite of our best intentions, a significant
proportion of patients treated with chemotherapy derive no clinical benefit, though all are exposed to
toxic and expensive therapeutic regimens. Therefore, there is a clear need to develop biomarkers that
can help predict which subset of patients will or will not benefit from these treatment regimens.
Currently there are no biomarkers available for determining response for oxaliplatin-based
chemotherapy (FOLFOX) which serves as standard-of-care for treating patients with advanced CRC.
 The significance of the current proposal stems from the huge unmet clinical need for the lack of
availability of a single established predictive/prognostic biomarker for stage III or IV CRC patients
treated with oxaliplatin-based chemotherapy, which is the standard-of-care treatment for such patients.
This gap in knowledge encouraged us to undertake systematic and comprehensive microRNA
(miRNA) biomarker discovery using next generation sequencing approaches, rigorous biomarker
prioritization and validation of candidate markers in multiple, independent patient cohorts, and potential
translation of some of these aberrantly methylated genes/loci as blood-based, noninvasive biomarkers.
To achieve these goals, we will pursue the following 3 specific aims: Aim #1: MiRNA biomarkers will be
“discovered” using miRNA-Seq in matched tumor and normal mucosa tissues from advanced CRC
patients with and without response to FOLFOX chemotherapy. Aim #2: Candidate miRNA biomarkers
will be “clinically validated” and their “performance evaluated” in primary tumor tissues from
independent cohorts of stage III (adjuvant treatment) and stage IV (palliative treatment) CRC patients
treated with FOLFOX. Aim #3: Determine the feasibility of translating “tissue-based” miRNAs into
“plasma-based” predictive biomarkers in metastatic CRC patients treated with FOLFOX.
 This project will be undertaken by a team of investigators who have longstanding experience and
track record in the field of cancer epigenetics and biomarker discovery. Our findings will have an
important clinical impact in identifying patients who will benefit from current chemotherapeutic
regimens, and those who are likely to experience adverse outcomes without benefit. The ability to
identify such biomarkers will help reduce the overall healthcare cost burden associated with such
treatments, and will provide a significant step forward into the era of personalized or precision medicine.
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## Key facts

- **NIH application ID:** 9983637
- **Project number:** 5R01CA202797-05
- **Recipient organization:** BECKMAN RESEARCH INSTITUTE/CITY OF HOPE
- **Principal Investigator:** Ajay Goel
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $405,048
- **Award type:** 5
- **Project period:** 2016-09-01 → 2023-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9983637

## Citation

> US National Institutes of Health, RePORTER application 9983637, MicroRNA Biomarkers for Determining Treatment Response in Colorectal Cancer (5R01CA202797-05). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/9983637. Licensed CC0.

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