# Focused ultrasound-enabled brain tumor liquid biopsy (FUS-LBx)

> **NIH NIH R01** · WASHINGTON UNIVERSITY · 2020 · $622,272

## Abstract

PROJECT SUMMARY/ABSTRACT
Approximately 700,000 patients in the United States have a primary central nervous system tumor, and ~200,000
new cases of brain metastases are diagnosed annually. The current approach for brain tumor diagnosis is mainly
through neuroimaging [e.g., magnetic resonance imaging (MRI)] to detect tumors with sufficient mass followed
by surgical resection or stereotactic biopsy for histologic confirmation of imaging findings. However, tissue
biopsies carry significant risk, are not feasible for repeated sampling to monitor the tumor progression and
treatment response, and may not be possible at all in surgically inaccessible tumor locations or medically
inoperable patients. Blood-based liquid biopsy offers a noninvasive approach to classify disease, guide therapy,
monitor treatment response, and unravel molecular mechanisms underlying the disease through the detection
of circulating tumor biomarkers (e.g., DNA, RNA, extracellular vesicles, and proteins shed by tumor cells). It has
transformed the clinical management of several cancers outside the brain. However, extending blood-based
liquid biopsies to brain cancer is challenging mainly because the blood-brain barrier (BBB) hinders the transfer
of tumor-derived biomarkers into the blood circulation system. To overcome this challenge, our group introduced
the focused ultrasound-enabled brain tumor liquid biopsy (FUS-LBx) technique for noninvasive and
spatially targeted molecular characterization of brain tumors. The central hypothesis is that FUS-mediated BBB
disruption opens “two-way trafficking” between brain and blood pool, thereby releasing brain biomarkers into the
blood circulation as well as allowing circulating drugs to enter the brain. Our long-term goal is to transform the
clinical management of patients with brain cancer by providing molecular signatures of the disease using
noninvasive FUS-LBx. The objective of this application is to obtain compelling preclinical evidence needed to
support future clinical translation of FUS-LBx. Our objective will be achieved by completing the following three
specific aims: (1) Evaluate the impact of FUS parameters on brain-tumor biomarker-release levels and safety
in a mouse brain tumor model; (2) Evaluate the impact of tumor variables on FUS-mediated brain-tumor
biomarker-release levels in mouse models of brain tumors; (3) Assess the feasibility and safety of FUS-LBx in a
pig brain tumor model. The proposed research contains three main innovations: (1) The hypothesis that FUS-
induced BBB disruption enables two-way trafficking between blood and brain opens a new research field; (2)
FUS-LBx is an innovative diagnostic tool and provides a new pathway to clinical translation of FUS technology;
(3) The pig brain tumor model that will be used in this study provides a large animal model that is critical for
obtaining unequivocal evidence to support the clinical translation of FUS-LBx. This project is significant
because this innovative t...

## Key facts

- **NIH application ID:** 10043944
- **Project number:** 1R01EB030102-01
- **Recipient organization:** WASHINGTON UNIVERSITY
- **Principal Investigator:** Hong Chen
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $622,272
- **Award type:** 1
- **Project period:** 2020-08-01 → 2024-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10043944

## Citation

> US National Institutes of Health, RePORTER application 10043944, Focused ultrasound-enabled brain tumor liquid biopsy (FUS-LBx) (1R01EB030102-01). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10043944. Licensed CC0.

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