The
findings of a major international trial to test 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.
UVA
neurosurgeon Jeff Elias, MD, and collaborators examined the benefits and risks
of using focused ultrasound to target an area deep within the brain called the
globus pallidus. The researchers wanted to see if the focused sound waves could
improve trial participants’ ability to move and reduce the unwanted shaking and
rigidity associated with Parkinson’s.
Of
the 69 patients who received the procedure in the randomized trial, almost 70%
responded to the treatment. Thirty-nine participants who received the procedure
continued to see significant benefits three months later, and 30 of those
assessed at the one-year mark continued to see benefits. The procedure, the
researchers conclude, could be particularly useful for patients who are
ineligible or unwilling to receive deep brain stimulation, a surgery that
implants electrodes deep in the brain to accomplish the same symptom-management
goals.
The
focused-ultrasound trial results 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 for the treatment of
Parkinson’s disease in 2021.
READ MORE: Selecting a health care provider
“This
study is promising for patients with Parkinson’s disease and other neurological
conditions. Ultrasound was precisely focused deep inside the brain to alter one
of the abnormal circuits of Parkinson’s disease,” Elias said. “But it is
important to understand that the treatment improved the neurological symptoms
of PD and did not alter its course. Ultimately, we hope to someday cure PD.”
About Focused Ultrasound
The focused ultrasound procedure focuses sound
waves inside the brain to disrupt faulty brain circuits, like how a magnifying
glass can focus light. Unlike deep brain stimulation, the minimally invasive
procedure does not require incisions or opening the skull. The procedure is
guided by magnetic resonance imaging (MRI), so doctors can pinpoint the exact
right spot in the brain before making any permanent changes.
Participants in the randomized Parkinson’s trial were assigned
to receive either the focused-ultrasound treatment or a harmless pretend – or
“sham” – procedure. In total, 69 received the actual treatment and 25 received
the sham procedure. The participants’ symptoms were then assessed using two
common rating scales, one for patients on medication and one for patients off
medication.
Sixty-nine percent of the procedure recipients improved by at least
three points on one or both of the rating scales. That’s compared with only 32%
(seven participants) in the control group. In the control group, significant
improvements were seen only in patients on medication.
Side effects reported by treatment recipients included trouble
speaking, difficulty walking and loss of taste. The loss of taste and
difficulty walking were mild and resolved on their own. One participant
continued to experience slurred speech a year after the procedure, the
researchers report.
Elias’ pioneering research into the use of focused ultrasound
for Parkinson’s is just the latest in his more than decade-long effort to tap
the vast potential of the technology. His prior work prompted the federal
Food and Drug Administration to approve focused ultrasound for the treatment of
essential tremor, a common movement disorder, in 2018. That approval
represented a turning point for the technology and helped spur additional
research at UVA and elsewhere.
Elias
and his UVA Health colleagues continue to explore the many possible
applications of focused ultrasound. For example, UVA Health last year joined
with the Charlottesville-based Focused Ultrasound Foundation – a longtime
supporter of UVA’s focused ultrasound research – to launch the world’s first
center devoted specifically to combining focused ultrasound with immunotherapy
to enhance the body’s ability to battle cancer. Other potential applications include using the technology to
open the brain’s natural protective barrier to allow, for the first time, the
targeted delivery of drugs for neurological 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 showcased a
procedure conducted by Elias and his team.
About
the Research
The
Parkinson’s research team consisted of Vibhor Krishna, Paul S. Fishman, Howard
M. Eisenberg, Michael Kaplitt, Gordon Baltuch, Jin Woo Chang, Wei-Chieh Chang, Raul
Martinez Fernandez, Marta del Alamo, Casey H. Halpern, Pejman Ghanouni, Roberto
Eleopra, Rees Cosgrove, Jorge Guridi, Ryder Gwinn, Pravin Khemani, Andres M.
Lozano, Nathan McDannold, Alfonso Fasano, Marius Constantinescu, Ilana
Schlesinger, Arif Dalvi and Elias. Elias is a consultant for Insightec, the
manufacturer of the focused ultrasound technology; a full list of the authors’
disclosures is included in the paper.
Financial
support for the Parkinson’s trial was provided by Insightec.
Other articles by HVP News Reporters