Focused Ultrasound Foundation Launches Five Preclinical Projects Targeting Glioblastoma, Liver Cancer, and Breast Cancer
Key Insights
Five new Foundation-funded preclinical projects were launched in Q2 2026 to advance focused ultrasound as a tool for improving therapeutic delivery and immune responses against difficult-to-treat tumors.
Two glioblastoma (search) projects will investigate focused ultrasound–mediated delivery of STING (search) agonists via nanocarriers and enhanced CAR T-cell therapy through blood-brain barrier opening.
A hepatocellular carcinoma (search) study will explore whether combining histotripsy with immunotherapy can trigger abscopal responses in untreated tumors.
Five new preclinical research projects funded by the Focused Ultrasound Foundation (search) have been initiated since April 2026, spanning the Foundation's Gene Therapy Program, Brain Tumors Program, and Immunotherapy Program. The studies aim to address key translational questions about how focused ultrasound can improve therapeutic delivery to the brain, strengthen immune responses against cancer, and enhance the effectiveness of therapies for difficult-to-treat tumors.
"Each of these projects is designed to answer a key translational question, helping move focused ultrasound closer to combination therapies and delivery strategies that can expand treatment options for patients," said Emily Whipple, PhD, the Foundation's Strategic Initiatives Director. "By generating both mechanistic insight and translational evidence, these studies are helping build the scientific foundation needed for future clinical development."
Optimizing Gene Delivery to the Brain
In the Gene Therapy Program, Rikke Kofoed, PhD, at Aarhus University Hospital leads a project focused on optimizing focused ultrasound–mediated gene delivery to the brain for clinical translation. While gene therapies may provide long-lasting benefits after a single treatment, the blood-brain barrier (BBB) remains a formidable obstacle, preventing many potentially beneficial treatments from reaching their target.
Previous studies in mice and large animals have demonstrated that focused ultrasound–mediated BBB opening (FUS BBBo) can significantly improve brain delivery of viral gene therapy after systemic administration. This project will investigate whether the timing of adeno-associated virus administration relative to FUS BBBo affects gene delivery in the mouse brain. The team will assess how timing influences delivery efficiency, which cell types are transduced, and how far the therapy spreads from the site of entry.
Advancing Immunotherapy for Glioblastoma
Two projects in the Brain Tumors Program target glioblastoma (search), an aggressive brain cancer with limited treatment options largely due to the BBB's restrictive nature.
Kristin Huntoon, DO, PhD, at the University of Arizona is leading a study on targeted immunotherapy using transcranial focused ultrasound–mediated delivery of STING (search) agonists via nanocarriers. The approach employs STING-laden Acousto-NanoDroplets (STANDs), which, when activated by focused ultrasound, are designed to simultaneously open the BBB and release a STING agonist (search) at the tumor site. This compound activates a natural immune alarm pathway, and the team will study whether the approach can help immune cells recognize and attack the tumor while limiting adverse effects.
A second glioblastoma (search) project, led by Jenny Munson, PhD, at Virginia Tech and funded through the Commonwealth of Virginia as a result of the Foundation's advocacy efforts, will test whether focused ultrasound can improve CAR T-cell delivery to glioblastoma in two ways: by improving the distribution of locally delivered CAR T-cells in tumor tissue, and by opening the BBB to help intravenously infused CAR T cells enter the brain. CAR T-cell therapy has been successful in treating some cancers, but its application in brain tumors remains challenging due to the BBB.
Harnessing Immune Responses in Liver and Breast Cancers
In the Immunotherapy Program, Tejaswi Worlikar, PhD, at the University of Michigan is investigating histotripsy-induced local and abscopal responses in hepatocellular carcinoma (search). Nearly two-thirds of patients with liver cancer have more than one tumor, and it is often not possible to directly treat every tumor. Histotripsy breaks apart tumor tissue without heat, which may release signals that help "wake up" the immune system. The team will test whether combining histotripsy with immunotherapy can help treat both the tumor targeted by focused ultrasound and other liver tumors that are not treated directly.
Timothy Bullock, PhD, at the University of Virginia leads a project—also funded through the Commonwealth of Virginia—exploring whether drugs that improve tumor blood flow can help the immune system respond more effectively after focused ultrasound treatment. Using mouse models of breast cancer (search), the team will test whether combining focused ultrasound thermal ablation with blood vessel–normalizing drugs and immunotherapy can improve treatment responses. Immunotherapies have produced meaningful results for many patients but do not work for everyone, including many with breast cancer, potentially because abnormal tumor blood vessels limit immune cell access.
Building the Foundation for Clinical Translation
The five projects collectively address critical barriers in oncology: the blood-brain barrier in brain tumors, immune exclusion in breast cancer (search), and multifocal disease in liver cancer. By generating both mechanistic understanding and translational evidence, these studies aim to establish the scientific groundwork needed for future clinical trials evaluating focused ultrasound as a component of combination treatment strategies.