- The Foundation’s Research Awards Program initiated eight preclinical studies in the second quarter.
- The projects span a wide range of mechanisms, including immunotherapy, neuromodulation, and gene therapy.
- Researchers will study diseases such as glioblastoma, neuroblastoma, opioid addiction, Parkinson’s disease, and spinal muscular atrophy.
The Foundation’s Research Awards Program initiated eight new preclinical studies in the second quarter of 2022. The funded projects span a wide range of mechanisms, including immunotherapy, neuromodulation, and gene therapy. Researchers will study diseases such as glioblastoma, neuroblastoma, opioid addiction, Parkinson’s disease, and spinal muscular atrophy. A veterinary study will enroll cats with urinary tract stones.
Each newly initiated project is listed below, along with the name of the principal investigator(s) and a brief project description.
Malignant Brain Tumors
Preclinical Study of the Potential of Sonosensitizer-Mediated Sonodynamic Therapy on Malignant Brain Tumor Treatment led by Kuo-Chen Wei, MD, at New Taipei City Hospital
This project seeks to replicate results from a preclinical sonodynamic therapy study conducted at the University of Virginia. An additional aim is to develop a novel noninvasive brain tumor treatment system that will be beneficial to patients with malignant brain tumors.
The Use of Monodisperse Size-Isolated Microbubbles for Improving the Safety and Efficacy of Chemotherapeutics in Neuroblastoma Treatment led by Jameel Feshitan, PhD, at Advanced Microbubbles
This project will compare monodisperse microbubbles with Definity and SonoVue microbubbles for enhancing intertumoral drug penetration and distribution of chemotherapeutics in a neuroblastoma mouse tumor model.
Focused Ultrasound Blood-Brain Barrier (BBB) Opening to Halt Nigrostriatal Degeneration led by José Obeso, MD, PhD, at Centro Integral de Neurociencias HM Cinac in Madrid, Spain
This translational study seeks to define nigrostriatal disease-modifying therapies that may be applied using MRI-guided focused ultrasound BBB opening in non-human primate models of Parkinson’s disease, with the ultimate goal of designing a pilot study in early Parkinson’s patients.
Development of MRI-FUS-PET led by Stuart Berr, PhD, at the University of Virginia Focused Ultrasound Immunotherapy Center
This project seeks to determine whether positron emission tomography (PET) can be integrated into an MR-guided focused ultrasound system and used in real time. After installation and testing, the team would will employ the combined system to deliver a radiolabeled antibody to a mouse model of glioma using focused ultrasound with microbubbles to open the blood-brain barrier.
Focused Ultrasound Delivery of miR-142-3p led by Roger Abounader, MD, PhD, at the University of Virginia Focused Ultrasound Immunotherapy Center
This project will investigate whether miR-142-3p (a master regulatory tumor suppressive micro RNA that targets many genes, including immune modulator genes and the MGMT gene) might be a potent immunomodulatory and temozolomide-sensitizing therapeutic agent for the treatment of glioblastoma. Temozolomide is a type of chemotherapy used to treat glioblastoma.
Spatial Interrogation of the Immune-Cell Co-Localization Associated with High Intensity Focused Ultrasound–Induced Lesions led by Awndre Gamache, PhD, and Timothy Bullock, PhD, at the University of Virginia Focused Ultrasound Immunotherapy Center
This project will investigate whether focused ultrasound–induced immunogenic cell death promotes co-localization of activated dendritic cells and tumor infiltrating lymphocytes. Secondly, it will determine whether sparse-scan focused ultrasound treatments facilitate immuno-modulation.
Focused Ultrasound–Mediated Gene Therapy for Treating Spinal Muscular Atrophy with Spatiotemporal Precision led by Guosong Hong, PhD, at Stanford University School of Medicine
This project will investigate whether focused ultrasound can offer a noninvasive means to perform in vivo gene editing with spatiotemporal precision, thus affording organ- and region-specific therapeutics for treating spinal muscular atrophy with minimal tissue damage.
Focused Ultrasound for Neuromodulation in the Treatment of Opioid Addiction led by Linda Chang, MD, MS, and Victor Frenkel, MD, PhD, at the University of Maryland School of Medicine
This project seeks to develop a novel approach for treating opioid addiction using MR-guided, pulsed, low intensity focused ultrasound (LIFU) targeting of the nucleus accumbens, a key reward-seeking structure in the brain. The team will measure whether extended effect, plastic neuromodulation by MR-guided LIFU can reduce drug self-administration in a rat model.
Evaluation of a Veterinary Focused Ultrasound Device to Noninvasively Fragment Feline Urinary Tract Stones led by Adam Maxwell, PhD, at the University of Washington
This project will study the safety and efficacy of using burst wave lithotripsy to treat urinary tract stones in companion animals (cats).