Charlottesville, VA, New York, NY, February 9, 2023 – The Focused Ultrasound Foundation and The Michael J. Fox Foundation for Parkinson’s Research (MJFF) have established a partnership to explore how focused ultrasound can impact the treatment of Parkinson’s disease.
For more than 15 years, the Focused Ultrasound Foundation has been dedicated to advancing the development and adoption of focused ultrasound. Since its inception, one of the Foundation’s priorities has been brain research, and movement disorders like Parkinson’s disease were among the first vanguard applications.
Likewise, MJFF was established in 2000 with the goal of finding a cure for Parkinson’s disease, and today it is the world’s largest non-profit funder of Parkinson’s drug development.
Focused Ultrasound: An Established Therapy for Parkinson’s
In 2018, MR-guided focused ultrasound earned US Food and Drug Administration (FDA) approval to treat tremor-dominant Parkinson’s disease using thermal ablation. This approval was expanded in 2021 to include patients with advanced Parkinson’s disease suffering with mobility, rigidity, or dyskinesia symptoms. MJFF and the Focused Ultrasound Foundation contributed funding for the initial research that led to these approvals.
It is important to note that this technique only offers relief from the debilitating symptoms of Parkinson’s disease and does not address the underlying cause of the disease.
A New Approach
Now, the two organizations have teamed up to fund a clinical trial investigating focused ultrasound’s ability to enhance a brain tissue sampling technique – called liquid biopsy – in patients with Parkinson’s disease.
The study will evaluate safety and preliminary efficacy of using Insightec’s Exablate Neuro focused ultrasound device to disrupt the blood-brain barrier (BBB), allowing proteins related to Parkinson’s disease to escape the brain and be detected in the peripheral blood.
Liquid biopsies analyze the molecular profile of tissue through a simple blood test. This technique can be challenging in the brain because of the BBB, a protective layer of tightly joined cells that line the blood vessels in the brain. The BBB is beneficial in that it prevents harmful substances from entering the brain. Unfortunately, it also inhibits the movement of molecular biomarkers from the brain into the bloodstream, limiting our ability to observe changes and identify potential biomarkers.
In this 10-participant trial, researchers will obtain blood samples before the procedure to establish a baseline and then at 10 and 60 minutes after focused ultrasound-induced BBB disruption. Based on data from preclinical studies, researchers hypothesize that focused ultrasound will enable a higher concentration of Parkinson’s disease-related biomarkers to enter the bloodstream. The ability to recognize and track these biomarkers will make it easier to diagnose, monitor, and develop novel therapies to address the disease.
“MJFF is proud to support innovative therapies such as focused ultrasound to help treat symptoms of Parkinson’s disease,” said Bradford Casey, PhD, MJFF’s senior associate director of discovery research. “We are hopeful that this partnership will advance clinical learnings and track disease progression to provide better treatments.”
The study is being led by Nora Vanegas, MD, associate professor of neurology and director of neuromodulation research at Baylor College of Medicine in Houston.
“This study may help develop a non-surgical method to identify disease-specific biomarkers for early diagnosis and precision follow up of Parkinson’s disease,” said Dr. Vanegas. “Monitoring the progression of Parkinson’s disease more accurately will improve our ability to provide better treatments.”
“We are honored to partner with MJFF on this clinical trial to explore a new way in which focused ultrasound could positively impact patients with Parkinson’s disease,” said Focused Ultrasound Foundation Chairman Neal F. Kassell, MD. “The Foundation is proud to have been a key driver – along with MJFF and others – in establishing focused ultrasound as a treatment for the symptoms of Parkinson’s disease, and we are eager to investigate how the technology can be used to aid in diagnosis and tracking of disease progression in response to potential therapies targeting the underlying disease.”
About Focused Ultrasound
Focused ultrasound uses ultrasound energy guided by real-time imaging to treat tissue deep in the body without incisions or radiation. It is FDA approved in the United States to treat essential tremor, tremor-dominant Parkinson’s disease, uterine fibroids, pain from bone metastases, osteoid osteoma, and the prostate. Dozens of additional indications are approved outside of the US. The technology is in various stages of research and development for more than 170 diseases, including Alzheimer’s disease and tumors of the brain, liver, breast, and pancreas.
About the Focused Ultrasound Foundation
The Focused Ultrasound Foundation was created to improve the lives of millions of people worldwide by accelerating the development of this noninvasive technology. The Foundation works to clear the path to global adoption by organizing and funding research, fostering collaboration, and building awareness among patients and professionals. Since its establishment in 2006, the Foundation has become the largest nongovernmental source of funding for focused ultrasound research.