Pioneering focused ultrasound trial shows promising results for Parkinson’s patients

Results of a major international trial testing a high-tech, scalpel-free approach to treating movement problems caused by Parkinson’s disease have been published in the prestigious New England Journal of Medicine.

Jeff Elias, MD, pioneered the use of focused ultrasound to treat essential tremor and Parkinson’s disease. Image credit: UVA Health

UVA neurosurgeon Jeff Elias, MD, and collaborators examined the benefits and risks of using focused ultrasound to target a region deep within the brain called the globus pallidus. The researchers wanted to see if focused sound waves could improve the participants’ ability to move and reduce the unwanted shaking and stiffness associated with Parkinson’s disease.

Of the 69 patients who underwent the procedure in the randomized trial, approximately 70% responded to treatment. Thirty-nine participants who had the procedure continued to see significant benefits after three months, and 30 of those assessed at one year continued to see benefits. The researchers concluded that this procedure may be especially useful for patients who are not eligible or unwilling to receive deep brain stimulation, which is surgery that implants electrodes deep into the brain to achieve the same goals as symptom management.

The results of the focused ultrasound trial were shared with the Federal Food and Drug Administration prior to publication and were an important consideration in the agency’s decision to expand approval of the technology to treat Parkinson’s disease in 2021.

This study is promising for patients with Parkinson’s disease and other neurological conditions. The ultrasound was precisely focused deep within the brain to alter one of the abnormal circuits in Parkinson’s disease. But it is important to understand that the treatment improved the neurological symptoms of Parkinson’s disease and did not alter its course. Ultimately, we hope to cure Parkinson’s disease one day.”

Jeff Elias, MD, UVA neurosurgeon

About focused ultrasound

The focused ultrasound procedure focuses sound waves inside the brain to disrupt faulty brain circuits, much like how a magnifying glass can focus light. Unlike deep brain stimulation, the minimally invasive procedure does not require incisions or opening of the skull. The procedure is guided by a magnetic resonance imaging (MRI) scan, so doctors can locate exactly the right spot in the brain before making any permanent changes.

Participants in the randomized Parkinson’s disease trial were assigned to receive focused ultrasound therapy or a harmless imaginary procedure – or a ‘sham’ procedure. In all, 69 received the real treatment and 25 received the placebo procedure. The participants’ symptoms were then assessed using two common scales, one for patients taking medications and one for patients not taking medications.

Sixty-nine percent of recipients of the procedure improved by at least three points on one or both rating scales. This compared to only 32% (seven participants) in the control group. In the control group, significant improvements were only seen in patients taking the medication.

Side effects reported by treatment recipients included difficulty speaking, difficulty walking, and loss of taste. Loss of taste and difficulty walking were mild and resolved on their own. Researchers reported that one participant continued to experience slurred speech a year after the procedure.

Elias’ pioneering research using focused ultrasound for Parkinson’s disease is the latest in his more than a decade-long effort to capitalize on the technology’s vast potential. His previous work led the Federal Food and Drug Administration to approve focused ultrasound for the treatment of essential tremor, a common movement disorder, in 2018. This approval marked a turning point for the technology and helped spur additional research at UVA and elsewhere.

Elias and his colleagues at UVA Health continue to explore the many possible applications of focused ultrasound. For example, last year UVA Health joined forces with the Charlottesville-based Focused Ultrasound Foundation—a longstanding supporter of UVA focused ultrasound research—to launch the world’s first center specifically dedicated to combining focused ultrasound with immunotherapy to enhance The body’s ability to fight cancer. Other potential applications include using the technology to open up the brain’s natural protective barrier to allow for the first time targeted delivery of drugs for neurodegenerative diseases such as Alzheimer’s.

UVA’s transformative work with focused ultrasound was recently highlighted around the world by CNN’s Sanjay Gupta in a story that featured a procedure performed by Elias and his team.

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The Parkinson’s disease research team consisted of Vipor Krishna, Paul S Fishman, Howard M Eisenberg, Michael Caplett, Gordon Baltush, Jin Wu Chang, Wei Chieh Chang, Raul Martinez Fernandez, Marta del Alamo, Casey H. Halpern, Pegman Gannon, Roberto Elliobra, and Rhys Cosgrove, Jorge Guridi, Ryder Gwen, Pravin Khimani, Andres M. Lozano, Nathan McDonald, Alfonso Fasano, Marius Constantinescu, Ilana Schlesinger, Arif Dalvey, and Elias. Elias is a consultant to Insightec, a manufacturer of focused ultrasound technology. A full list of authors’ disclosures is included in the paper.

Financial support for the Parkinson’s disease trial was provided by Insightec.