# Novel Image-guided, remote-controlled opening of the blood-brain barrier for systemic brain tumor therapy

> **NIH NIH P01** · WAKE FOREST UNIVERSITY HEALTH SCIENCES · 2021 · $281,901

## Abstract

Glioblastoma (GBM) is the most common and aggressive primary brain tumor, with a dismal prognosis. The
poor prognosis is a direct result of the fact that potentially efficacious therapeutics cannot access all primary
tumor and infiltrating GBM cells due to the blood-brain barrier (BBB) or blood-brain tumor barrier (BBTB). Our
central hypothesis is that we can use novel high-frequency ultrasound (HIFU)-based techniques to remotely
permeabilize the BBB and systemically delivery conventional chemotherapy and novel molecular targeted
therapeutics to GBM, especially infiltrating cells that reside in predominantly normal brain. In addition to
conventional HIFU techniques, we will use an innovative remote-controllable stem cell-based platform to allow
us to non-invasively activate engineered stem cells to produce specific cytokines at the time and site of our
choosing. To test these hypotheses, 2 specific aims will be performed: (Specific Aim 1) To demonstrate the
feasibility of using image-guided HIFU remote activation to artificially induce TNFα and permeabilize the BBB
and thereby allowing systemically administered therapeutics to access invasive GBM cells; (Specific Aim 2) To
demonstrate anti-GBM efficacy of novel molecular targeted therapeutics after systemic infusion and HIFU-
mediated BBB/BBTB focal permeability. Delivery of therapeutic agents through the BBB specifically to tumor
cells but not to the surrounding normal brain tissue offers hope against GBM, an invariably fatal disease. Thus,
this proposal has strong translational relevance.

## Key facts

- **NIH application ID:** 10220884
- **Project number:** 5P01CA207206-05
- **Recipient organization:** WAKE FOREST UNIVERSITY HEALTH SCIENCES
- **Principal Investigator:** Akiva Mintz
- **Activity code:** P01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $281,901
- **Award type:** 5
- **Project period:** 2017-08-17 → 2023-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10220884

## Citation

> US National Institutes of Health, RePORTER application 10220884, Novel Image-guided, remote-controlled opening of the blood-brain barrier for systemic brain tumor therapy (5P01CA207206-05). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10220884. Licensed CC0.

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