Research Awards Update: Six Preclinical Projects Initiated in the First Quarter of 2022


Key Points

  • The Foundation’s Research Awards Program has initiated six preclinical studies this year.
  • All funded projects this quarter are brain projects, ranging from improving treatment planning to treating brain metastases and delivering gene therapy for neurological disorders.

The Foundation’s Research Awards Program initiated six new preclinical studies in the first quarter of 2022. Projects are exploring the use of focused ultrasound to treat or improve treatment for various indications. Two projects focus on delivering gene therapy for neurological disorders. Two projects are directed toward treating brain metastases and leptomeninges. One of the metastatic projects is testing sonodynamic therapy in a model of melanoma that has spread to the brain. Each newly initiated project is listed below.

Improving Treatment Planning

Optimizing Relationships Between Skull Density and Acoustic Properties using Magnetic Resonance Temperature Imaging Obtained during Clinical Focused Ultrasound led by Nathan McDannold, PhD, at Brigham and Women’s Hospital.
This project with determine whether simulations of transcranial focused ultrasound procedures can improve treatment planning using thermometry imagery from patient treatments.

Development of a Clinically-Feasible Image Processing Platform for Diffusion MRI Tractography–Based Focused Ultrasound Targeting of Essential Tremor led by Jennifer McNab, PhD, at Stanford University School of Medicine.
This project will develop a simple, open-source graphical user interface to provide greater access to tractography-based targeting for focused ultrasound treatment of essential tremor.

Gene Therapy

Enhancing Delivery of Molecular Therapies to the Central Nervous System for Genetic Neurological Disorders led by Nicholas Todd, PhD, at Brigham and Women’s Hospital.
This project will investigate the use of modified-RNA therapy for reducing intracellular cholesterol accumulation.

Focused Ultrasound–Mediated Delivery of the Neurorestorative Gene Therapy, rAAV9.SIRT3-myc, as a Disease-Modifying Strategy in Parkinson’s Disease led by JoAnne Nash at the University of Toronto.
This project will evaluate focused ultrasound as an improved method of gene therapy delivery for Parkinson’s disease. Focused ultrasound delivery will be compared with a more widely used, more invasive, less safe gene delivery approach.

Brain Metastases and Leptomeninges

Focused Ultrasound–Mediated Leptomeninges Opening to Facilitate Targeted Drug Delivery in Leptomeningeal Brain Metastases led by Eun Jung Lee, MD, at Seoul National University Hospital.
This project will use focused ultrasound to open the blood–cerebral spinal fluid barrier to enable drug delivery to the leptomeninges with the goal of reducing tumor burden and improving survival.

Sonodynamic Therapy for Metastatic Melanoma: An In Vivo Study led by Jason Sheehan, MD, PhD, and Zhiyuan Xu, MD, at the University of Virginia.
This project seeks to determine whether 5-ALA–based sonodynamic therapy (SDT) can lead to a complete response in a syngeneic murine subcutaneous model of melanoma. It will also assess SDT’s mechanisms of action in this context, including immune system modulation.

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