Key Points
- The Foundation’s Research Awards Program recently initiated preclinical projects that use focused ultrasound to treat Huntington’s disease and benign and cancerous tumors.
- Learn how to apply for funding.
The Foundation is pleased to announce that five new preclinical studies were launched in the second quarter of 2024. Researchers are working on technical ways to improve the delivery of focused ultrasound and studying the use of the technology to treat Huntington’s disease, sarcoid tumors, osteosarcoma, and more.
“These five projects build our knowledge of key focused ultrasound mechanisms but, more importantly, they address unmet clinical needs in cancer and Huntington’s disease,” said Joe Kilroy, PhD, the Foundation’s associate director of research and education. “We are excited to support this research and look forward to the results.”
Each newly initiated project is listed below.
Gene Therapy Program (2)
Enhancing Central Nervous System Penetration of Systemic Genetic Therapy for Huntington’s Disease led by Robert Friedlander, MD, at the University of Pittsburgh Medical Center
Huntington’s disease (HD) is a fatal, untreatable neurodegenerative disease caused by a genetic mutation. The mutant gene generates toxic levels of the mutant huntingtin (mHTT) protein. The ideal HD therapy would be a systemically administered drug that selectively reduces mHTT levels in the brain and affected peripheral organs, including the heart and liver. The researchers have identified a novel compound that could potentially be a treatment for HD. For this preclinical project, they are seeking to determine whether focused ultrasound can increase brain penetration of this new compound and reduce mHTT.
Enhancing Targeted Delivery of Paclitaxel by Focused Ultrasound–Mediated Disruption of Plasma Protein Binding led by Seung-Schik Yoo, PhD, MBA, at Brigham and Women’s Hospital and Harvard Medical School
The bioavailability of many types of chemotherapy agents, including paclitaxel (PTX), is profoundly reduced when bound to plasma proteins. Because the application of acoustic pressure waves can temporarily unbind drug molecules from plasma proteins, researchers will test whether applying pulsed, low-intensity focused ultrasound to a preclinical ovarian cancer tumor model will unbind intravenously injected PTX from plasma albumin. The goal is to increase drug delivery to the tumor and enhance its tumoricidal effects without increasing systemic dose.
Veterinary Program (2)
Feasibility of Histotripsy as a Noninvasive Therapy for Sarcoid Tumors in Horses led by Sophie Bogers, BVSc, PhD, at the Virginia-Maryland College of Veterinary Medicine*
Sarcoids are the most common tumor that occurs in horses, and they can cause poor quality of life when they occur in certain locations because current treatment options are limited. These fibroblastic, wart-like skin lesions can be invasive, but they are not necessarily malignant. For this study, researchers will apply histotripsy to sarcoid tumor tissue while preserving the surrounding normal tissue. Researchers will also evaluate whether the application of histotripsy stimulates an immune response. If successful, the treatment may be translatable to skin tumors in canines and humans.
Furthering Advances in Histotripsy Ablation of Canine Appendicular Osteosarcoma by Joanne Tuohy, PhD, DVM, at the Virginia-Maryland College of Veterinary Medicine
Osteosarcoma is the most common primary bone cancer in people and dogs; it is a painful and devastating tumor that metastasizes to other parts of the body and thus far has not been responsive to cancer immunotherapies. An initial study demonstrated histotripsy’s ability to ablate osteosarcoma; now researchers will seek to optimize the histotripsy parameters to enable targeting of the entire tumor and further evaluate the long-term effects of histotripsy ablation on the immunological, functional, and quality-of-life outcomes of canine patients with osteosarcoma.
General Technical (1)
Accelerating the Boiling Histotripsy Method by Rapid Steering the Focus Axially During the Pulse Delivery led by Oleg Sapozhnikov, PhD, from the University of Washington
Boiling histotripsy (BH) is a high intensity focused ultrasound (HIFU) cavitation-based method for mechanically ablating tissue. BH has shown promise for diverse clinical applications and offers multiple advantages over HIFU thermal ablation, but its ablation rate is lower than that of thermal HIFU. For this project, researchers will test whether steering the HIFU focus position during BH pulse delivery can extend the cavitation cloud, thus extending its resulting tissue lesion. The team plans to optimize this new method of BH for accelerating mechanical tissue ablation and also determine the extent to which the volumetric rate of BH ablation can be accelerated.
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*This Virginia Tech project was funded by the Commonwealth of Virginia as a result of the Foundation’s advocacy efforts.