# A theranostic system for ultrasound-facilitated blood-brain barrier opening

> **NIH NIH R01** · COLUMBIA UNIVERSITY HEALTH SCIENCES · 2021 · $656,950

## Abstract

Today, over 35 million Americans are over the age of 65. Within the next 30 years this number is
likely to double, putting more and more people at increased risk of neurodegenerative disease.
Over the past several decades, numerous small- and large-molecule products have been
developed for treatment of neurodegenerative diseases with mixed success. When administered
systemically in vivo, the blood-brain barrier (BBB) inhibits their delivery to the regions affected by
those diseases. Safe and localized opening of the BBB has been proven to pose an equally
significant challenge. Focused Ultrasound (FUS), in conjunction with microbubbles, remains the
sole technique that can induce localized BBB opening noninvasively. Previously, our group has
demonstrated that neuroprotection and neurorestoration in the dopaminergic neurons in the
nigrostriatal pathway at the early stages of disease can be induced through ultrasound-mediated
drug delivery. In this study, we will develop and optimize a theranostic ultrasound system that can
simultaneously induce and map the BBB opening in mice and primates in vivo. As a secondary
objective, we will determine whether this new BBB opening systems are sufficient to induce BBB
at sufficient levels to induce neuroprotection and neurorestoration through gene delivery in mice
and NHP for the treatment of motor symptoms in Parkinsonian models associated with early-
stage PD. The multi-disciplinary team assembled encompasses all critical specialty areas such
as ultrasound and microbubble engineering, neuroscience as well as MRI, PET and fluorescence
brain imaging, behavioral testing and mouse model development as well as clinical specialty in
movement disorders.This study will thus determine whether theranostic FUS can have a more
widespread impact by being implemented in diagnostic imaging scanners and efficacious enough
for the treatment of early stage Parkinson’s in conjunction with gene delivery.

## Key facts

- **NIH application ID:** 10104490
- **Project number:** 5R01EB029338-02
- **Recipient organization:** COLUMBIA UNIVERSITY HEALTH SCIENCES
- **Principal Investigator:** Elisa E. Konofagou
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $656,950
- **Award type:** 5
- **Project period:** 2020-03-01 → 2023-12-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10104490

## Citation

> US National Institutes of Health, RePORTER application 10104490, A theranostic system for ultrasound-facilitated blood-brain barrier opening (5R01EB029338-02). Retrieved via AI Analytics 2026-05-21 from https://api.ai-analytics.org/grant/nih/10104490. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*