Ultrasound-guided gene delivery to brain tumors

Project Name: Ultrasound-guided gene delivery to brain tumors
Status: Active
Primary Site: University of Virginia Health System
Sponsor Organization: Focused Ultrasound Foundation
Funded Date: Aug 01, 2021
Funding Period: Aug 01, 2021 - Aug 01, 2022
Funding Program: Immunotherapy
Category: Pre-Clinical
Amount Awarded: $127,540.00


Antibody-based immunotherapy (e.g. check point inhibitors) has emerged as one of the most promising directions in clinical oncology.1 These agents and their synergistic combinations show promising pre-clinical efficacy in a wide range of incurable malignancies, including malignant brain tumors – one of the deadliest types of cancer known.2 Unfortunately, translation of most of these approaches remains difficult due to challenges in controlling the bio-disposition of antibody-based drugs.3 Owing to their unfavorable systemic biodistribution, high dose regimens are required to attain and maintain therapeutic drug concentrations in tumors.4, 5 Such regimens yield high off-target drug exposure and deleterious systemic toxicity, posing a difficult barrier to clinical translation.3, 6 We aim to develop a new approach for placing the bio-biodisposition of therapeutic antibodies under focused ultrasound control. This approach harnesses our newly developed messenger RNA (mRNA) nanoparticles, mRNAresp, that facilitate transfection via an ultrasound-controlled gating mechanism. Our strong preliminary data reveal that mRNAresp allow leveraging focused ultrasound to direct expression of mRNA-encoded proteins to specific tissue sites with minimal expression in off-target tissues. In this study, we will assess the applicability of this approach for directing expression of mRNA-encoded antibody therapeutics to brain tumors and determine the ensuing efficacy and toxicity. We expect to demonstrate that this approach has the capacity to offer anti-tumor efficacy that is comparable to standard protein-based antibody treatments, while significantly reducing off-target toxicity. Successful completion of the project goals will yield a clinically viable strategy to unlock the full potential of therapeutic antibodies for combating refractory malignancies.

Contact Roles


No reports found.

The Focused Ultrasound Foundation Newsletter

Our newsletter delivers updates for clinicians, researchers and patients. Sign up and stay on top of the rapid advancements of this innovative medical technology.

Sign Up  Read the Latest Issue

Read The Tumor  by John Grisham

From John Grisham, comes a story where today’s medical fiction could become tomorrow’s lifesaving reality.

Download a Free eBook or Audiobook

Contact Details

Call Us
Find Us
Focused Ultrasound Foundation
1230 Cedars Court, Suite 206 Charlottesville, VA 22903
Whitelist Instructions