Key Points
- The Foundation’s Research Awards Program has initiated eight preclinical studies this year.
- Along with improving brain treatments, the funded projects use focused ultrasound to address Parkinson’s disease, pediatric neuroblastoma, tissue healing, cancer, and more.
The Foundation’s Research Awards Program initiated eight new preclinical studies in the first quarter of 2023. Along with improving brain treatments, the funded projects use focused ultrasound to address Parkinson’s disease (with gene therapy), pediatric neuroblastoma, breast cancer, liver trauma, liver cancer, uterine diseases, and tissue healing. One of the brain projects will be led by a next-generation scholar.
“With so many new projects getting underway in the first quarter, it is encouraging to see such a strong start to 2023,” said Matt Eames, PhD, the Foundation’s Director of Extramural Research. “This cohort includes one of our first active preclinical gene therapy projects, which is using sonogenetics to augment stem cell therapy for Parkinson’s disease.”
Each newly initiated project is listed below.
Gene Therapy
Sonogenetics-Enabled Spatial and Temporal Expression of Transcription Factors for Direct Programming of Human Stem Cells led by Yun Jing, PhD, at Pennsylvania State University
In this project, researchers will develop a new ultrasound-based (sonogenetic) approach for addressing the current limitations of stem cell therapy for treating Parkinson’s disease.
Brain
Investigation of Pulse Sequences for Nanodroplet-Enhanced Focused Ultrasound Mechanical Ablation in the Rat Brain led by Swanee Douglas at Brigham and Women’s Hospital*
In this next-generation scholar project, researchers will design, prepare, and perform preclinical experiments with a clinical focused ultrasound device for specialized brain applications. They will use MRI for treatment guidance and assessing treatment response. Swanee Douglas is an undergraduate student at the University of Toronto. She received the Foundation’s 2022 Global Intern Award.
Immunotherapy
Ultrasound-Enhanced CAR-T Cell Immunotherapy of Pediatric Neuroblastoma led by Gail ter Haar, PhD, at the Institute of Cancer Research*
This project will determine whether histotripsy exposure can improve engineered CAR-T cell uptake and immunotherapy efficacy in neuroblastoma. Engineered CAR-T cells are effective against blood cancers but lack efficacy in soft tissue tumors, in part due to their low uptake. Although concomitantly administered vascular permeabilizing drugs improve tumor uptake, they remain suboptimal. Histotripsy may enhance uptake sufficiently to provide an effective treatment for pediatric neuroblastoma.
Biomechanical Modulation of Inflammation with Focused Ultrasound for Breast Cancer Therapy led by Charles Caskey, PhD, at Vanderbilt University
To address a knowledge gap about how pulsed focused ultrasound modulates the immune system in cancer therapy, researchers will characterize the effects of focused ultrasound immunomodulation on healthy tissue and the spleen in a mouse model.
Body
Focused Ultrasound–Induced Hemostasis for the Treatment of Liver Lacerations (in Swine) led by John Eisenbrey, PhD, at Thomas Jefferson University Hospital
In this project, which began in September 2022, researchers will detect active bleeding from liver lacerations with contrast-enhanced ultrasound (CEUS) and then stop the bleeding with high-intensity focused ultrasound (HIFU). The liver is commonly injured from trauma.
Focused Ultrasound Targeting for Interstitial Image-Guided Conformal Destruction of Tumor Volumes led by Apatou N’Djin, PhD, at Inserm LabTAU*
For this project, researchers are developing new focused ultrasound equipment for the treatment of hepatocellular carcinoma (liver cancer). They are customizing a current focused ultrasound system with an innovative, image-guided, miniaturized energy delivery device to access hard-to-reach liver cancers.
Noninvasive High-Intensity Focused Ultrasound Treatment of Functional Uterine Diseases led by David Melodelima, PhD, and Benoît de la Fourniere, MD, at Inserm LabTAU*
To improve the efficiency of noninvasive focused ultrasound treatment of fibroids and adenomyosis, researchers will design and build a toroidal transducer that could reduce recovery time, scarring, and the need for anesthesia during the procedure.
Low Intensity Focused Ultrasound (LoFU) for the Noninvasive Triggering of Silk Scaffold Degradation led by Rosalyn Abbott-Beauregard, PhD, at Carnegie Mellon University
To determine whether LoFU can assist with wound healing and tissue growth, researchers will closely monitor the healing stages of individual patients and then apply LoFU on-demand to trigger certain healing processes.
*Focused Ultrasound Center of Excellence