# Phase I trial of intravenous vesicular stomatitis virus for treatment of metastatic endometrial cancer

> **NIH NIH R01** · MAYO CLINIC ROCHESTER · 2020 · $543,708

## Abstract

Here we seek to understand the effect of systemically administered Vesicular Stomatitis Virus (VSV) on
humans and against metastatic endometrial cancer. This project includes the first-in-human clinical trial of
VSV engineered to encode human interferon β (hIFNβ) and the sodium iodide symporter (NIS). hIFNβ codes
for interferon (IFN) which protects normal cells from VSV effects and NIS enables tumor cells infected with the
virus to concentrate various iodine isotopes which can allow for imaging of tumors infected with the virus. The
virus being tested in this project is VSV-hIFNβ-NIS. We selected VSV as the platform virus for this study
because: 1) VSV is a non-lethal, non-human (livestock) virus with mild, if any, symptoms in natural human
infections, 2) VSV exposure in the United States is rare with <5% of the population being seropositive, 3) VSV
has been safely administered to >7500 humans as the platform virus for HIV and Ebola virus vaccine
development, 4) preclinical studies have shown that VSV potently and rapidly kills endometrial cancer cells and
tumors, 5) advanced stage and high grade endometrial cancers have been shown to express receptors from
the low density lipoprotein receptor (LDLR) family that VSV utilizes for cell infection, 6) mouse and canine
studies have established the safety and maximum tolerated dose (MTD) of VSV-hIFNβ-NIS in those mammals,
7) mouse studies have shown that VSV-hIFNβ-NIS localizes to cancer and can be detected via SPECT/CT
imaging, and 8) a phase I trial of a similar virus strain, VSV-hIFNβ, is currently being performed in human
subjects. This project has incorporated real-time assessments of toxicity, tumor infectivity, and systemic
immune responses to the virus and against endometrial cancer. The overarching goal is to generate a
comprehensive understanding of the impact of systemic administration of the virus on the immunocompetent
human and determine tumor-specific effects of virus infection. The three specific aims to be investigated in this
project are: 1. Determine the safety, toxicity and tumor specificity of VSV-hIFNβ-NIS when administered
intravenously to patients with metastatic endometrial cancer. 2. To determine VSV-hIFNβ-NIS replication
pharmacokinetics, and shedding after virus administration and perform RNAseq and exome sequencing on
tumor and normal tissues to test a novel gene panel as a biomarker for tumor response to the virus. 3.
Determine the impact of intravenous VSV-hIFNβ-NIS on adaptive immune responses against the virus,
endometrial cancer antigens, and immunosuppressive mediators.

## Key facts

- **NIH application ID:** 10000856
- **Project number:** 5R01CA207240-04
- **Recipient organization:** MAYO CLINIC ROCHESTER
- **Principal Investigator:** Jamie N Bakkum-Gamez
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $543,708
- **Award type:** 5
- **Project period:** 2017-05-04 → 2022-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10000856

## Citation

> US National Institutes of Health, RePORTER application 10000856, Phase I trial of intravenous vesicular stomatitis virus for treatment of metastatic endometrial cancer (5R01CA207240-04). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10000856. Licensed CC0.

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