FDA approved for both Tremor Dominant Parkinson’s Disease and Dyskinesia
Focused Ultrasound Therapy
Focused ultrasound is an early-stage, noninvasive, therapeutic technology with the potential to improve the quality of life and decrease the cost of care for patients with Parkinson’s disease. This novel technology focuses beams of ultrasonic energy precisely and accurately on targets deep in the brain without damaging surrounding normal tissue.
How it Works
Where the beams converge, focused ultrasound produces a variety of therapeutic effects enabling Parkinson’s disease to be treated without surgery. For movement disorders, the mechanism is ablation (thermal disruption of the tissue) that can be aimed at several different treatment targets. For the preclinical work in treating the underlying cause, the mechanisms can be temporarily disrupting the blood-brain barrier (BBB), which will allow desired therapeutics the ability to enter the brain. Opening the BBB also can enable undesired materials to more easily depart from the brain.
Currently, there is no cure for Parkinson’s and the major options for treatment of motor symptoms include drug therapy and invasive surgery (e.g. deep brain stimulation, radiofrequency lesioning).
For certain patients, focused ultrasound could provide a noninvasive alternative to surgery with less risk of complications and lower cost. In the long term, focused ultrasound has the potential to treat the underlying disease pathology and prevent progression and/or restore function.
Relief of Motor Symptoms
The FDA has approved the use of focused ultrasound for treatment of tremor-dominated Parkinson’s disease. See more in the regulation and approval section, below.
Focused ultrasound has the potential to achieve symptomatic relief by making thermal lesions deep in the brain to interrupt circuits involved with tremor and dyskinesia. Symptoms and targets being assessed for treatment using focused ultrasound include:
- Parkinsonian tremor – target in the thalamus (thalamotomy)
- Parkinsonian dyskinesia – target in the globus pallidus (pallidotomy) or subthalamic nucleus
- Parkinsonian tremor, akinesia or dyskinesia – target in the pallidothalamic tract
At this time, focused ultrasound is only being assessed to treat one side of the brain, so it will affect tremor or dyskinesia unilaterally. Studies are being organized to assess the possibility of treating patients bilaterally, and one is included below.
- Focused ultrasound is non-invasive – no incisions, holes in the skull, electrodes in the brain – and therefore has reduced risk for infection and blood clots.
- Precise targeting minimizes damage to non-targeted healthy brain.
- Compared to deep brain stimulation, focused ultrasound is a single procedure, and does not require subsequent procedures/visits to replace batteries, repair broken wires, or adjust simulator settings. It also does not involve the collateral damage to healthy tissue or the risk of infections associated with implanting a foreign body.
Treating Underlying Pathology
Preclinical studies suggest focused ultrasound’s potential to restore function in Parkinson’s models. Focused ultrasound can temporarily open the blood-brain barrier (BBB) to improve the delivery of:
- Genes, growth factors, stem cells, other neuroprotective and/or neurorestorative drugs
- Anti-alpha synuclein antibodies
In October 2018, the Foundation had a multi-disciplinary workshop that aimed at using BBB opening in conjunction with potentially curative agents, to decide what research approaches have the most potential. Read the workshop summary >
There are several recent trials that assess the feasibility, safety, and preliminary efficacy of focused ultrasound to treat different symptoms of Parkinson’s. See a list of all Parkinson's clinical trials >
Parkinson’s dyskinesia (unilateral pallidotomy)
This international pivotal trial has completed enrollment of patients.
Parkinson’s disease (Staged unilateral or bilateral pallidothalamic tractotomy [PTT])
This trial is currently recruiting patients at multiple locations. Please note that patients who have had focused ultrasound treatments for tremor (at the VIM nucleus) or dyskinesia (at the GPi) are not eligible to have the other side treated in this clinical trial. The staged bilateral treatment for this clinical trial is only for patients who had the PTT on the first side.
Parkinson’s disease (Staged bilateral pallidothalamic tractotomy)
This trial is currently recruiting patients in Tokyo.
Regulatory Approval & Reimbursement
The Exablate system manufactured by Insightec is approved in Europe and in the US for treating tremor-dominant Parkinson's disease. In November 2021, it also earned approval in the US for treating additional movement disorders associated with Parkinson's disease, including mobility, rigidity and dyskinesia. We recommend that patients talk with the treatment site for questions about insurance coverage under Medicare, as some treatment sites are now being reimbursed by Medicare. Most commercial companies are not currently covering this procedure.
Preclinical Laboratory Studies
Preclinical studies suggest focused ultrasound’s potential to restore function in Parkinson’s models. Active studies are assessing focused ultrasound-induced BBB opening to improve the delivery of:
- Genes, growth factors, stem cells, other neuroprotective and/or neurorestorative drugs (University of Virginia/Johns Hopkins University; University of Maryland; Columbia University; Chang Gung University in Taiwan; University of Toronto)
- Anti-alpha synuclein antibodies (Columbia University; HM Puerta del Sur in Madrid/Institut Langevin in Paris)
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Click here for additional references on PubMed.
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