The National Institute of Neurological Disorders and Stroke (NINDS) recently notified Rich Price, PhD, at the University of Virginia, and Jung Soo Suk, PhD, at Johns Hopkins University (JHU) that they received a 5-year, $2.5 million “R01” research grant for their project titled “Innovative systemic gene therapy for treating Parkinson's disease.”
Because clinical trials for delivering gene therapy to patients with Parkinson’s disease have failed due to inefficient gene transfer to the diseased areas in the brain, the collaborative research team that also included Justin Hanes, PhD, from JHU, conducted a pilot study (funded by the Focused Ultrasound Foundation) to show that focused ultrasound could effectively open the blood-brain barrier (BBB) and release a therapeutic level of gene therapy from nanoparticles in a preclinical model of Parkinson’s disease.
Now, to bridge the technology to clinical translation, researchers at the two laboratories will use the NINDS funding to refine their delivery strategy in preclinical models that are even more similar to humans. If successful, the approach could allow earlier stage Parkinson’s patients who are more likely to respond to therapy to be included in clinical trials. The method may also be applicable for treating other neurological disorders.
"Focused ultrasound constitutes a simple, efficient, and targeted means to overcome the BBB by systemically administered nanoparticles, such as gene vectors," said Dr. Jung Soo Suk. "Unlike other methods that involve universal BBB opening and/or complex nanoparticle formulation, focused ultrasound provides a clear regulatory path for clinical development of systemic gene therapy of brain diseases, including but not limited to Parkinson's disease."
“One of the main goals of the Foundation’s funding awards is to show proof of principle, concepts, or ideas,” said Matt Eames, PhD, the Foundation’s External Awards Manger. “This type of follow-on funding shows how our small initial investment in a project can be amplified many times over. We congratulate Dr. Price, Dr. Hanes, and Dr. Suk on this impressive research award.”