FOCUSED ULTRASOUND

The US Food and Drug Administration has approved focused ultrasound for Parkinson’s. Patients can seek commercial treatment at participating sites,

Focused Ultrasound Therapy – Focused ultrasound is an early-stage, non-invasive, 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 leave the brain.

Advantages – 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 or akinesia – 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. 

Advantages 

• 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.

This article can be seen in its entirety at FusFoundation.org