Focused Ultrasound Therapy for Brain Disorders

Summary: Transcranial pulsed stimulation (TPS) and other focused ultrasound techniques can non-invasively target and modulate deep brain regions, opening new treatment possibilities for a wide range of neurological and psychiatric disorders.

Source: University of Vienna

Targeted ultrasound is emerging as more than an imaging tool: focused acoustic pulses can now be used to treat brain disorders with precision, reaching deep structures without opening the skull.

In recent years, several breakthrough ultrasound-based approaches for brain therapy have been developed by research groups in Toronto and at MedUni Vienna. These methods use precisely directed acoustic energy to either stimulate or suppress neural activity, to perform non-invasive focal lesioning, or to transiently open the blood–brain barrier for localized drug or gene delivery.

The Vienna-developed method—transcranial pulsed stimulation (TPS)—aims to improve brain function by selectively activating neurons that remain viable. This targeted neuromodulation may benefit patients with neurodegenerative and neuropsychiatric conditions such as Alzheimer’s disease, Parkinson’s disease, stroke-related deficits, multiple sclerosis, and certain chronic pain syndromes like neuralgia.

A joint review by investigators at MedUni Vienna and the University of Toronto, published in the journal Advanced Science, summarizes recent technical advances and patient results, and indicates that these ultrasound innovations are approaching broad clinical application.

New technological refinements now enable three distinct clinical strategies with focused ultrasound: precise non-invasive surgery for selected targets, focal delivery of therapies or genes via controlled opening of the blood–brain barrier, and highly targeted neuromodulation to restore dysfunctional circuitry. Each approach addresses different clinical needs and, together, they expand options for treating disorders that were previously difficult to reach safely.

Roland Beisteiner, who led the TPS development at the Department of Neurology at MedUni Vienna and Vienna General Hospital, emphasizes that these ultrasound methods are not intended to replace established therapies but to complement them. “The techniques developed in Vienna and Toronto are innovative additional options that can augment existing treatments,” Beisteiner says. Published patient data indicate that transcranial ultrasound approaches are safe and increasingly ready for clinical use, with the Viennese TPS method showing minimal side effects in published studies.

Viennese TPS: broad clinical roll-out in sight

The TPS neural stimulation technique, developed by an international consortium led from Vienna, was featured as a cover article in a leading scientific journal in early 2020. In an initial pilot study, patients with Alzheimer’s disease experienced measurable and sustained improvements lasting up to three months after treatment.

Clinical deployment of TPS is underway in specialized centers, but Beisteiner cautions that broader adoption requires clinicians with specific neurological expertise and familiarity with functional brain mapping. “This therapeutic approach is an evolving scientific field and requires practitioners to understand both the neurological context and the methodological aspects of ultrasound neuromodulation,” he explains.

Beyond conditions previously studied with less focal stimulation methods—like Alzheimer’s, Parkinson’s, stroke, multiple sclerosis, and neuralgia—TPS may enable novel indications because it uniquely allows non-invasive activation of deep brain regions. Any disorder where restoring impaired function by stimulating surviving neurons is a viable strategy could be a candidate for TPS. Notably, TPS has already obtained CE certification for the treatment of Alzheimer’s disease.

Ultrasound techniques from Toronto: different technique, different targets

Researchers at the University of Toronto, led clinically by Andres Lozano, have advanced complementary ultrasound technologies. One approach performs highly focused non-invasive lesioning and is already approved for managing essential tremor and tremor-dominant Parkinson’s disease. This method selectively deactivates overactive neurons without opening the skull, offering an alternative to more invasive surgeries.

This shows a brain
Highly focused ultrasonic waves can be applied for non-invasive surgery, focal therapeutic delivery, or targeted modulation of neural networks in various brain diseases. Image is in the public domain

A third Toronto-derived innovation uses focused ultrasound to transiently and locally open the blood–brain barrier, making it possible to deliver drugs, antibodies, or gene vectors directly to affected brain regions. This development addresses a longstanding challenge in neurology: many effective therapeutics cannot reach their intended targets because of the brain’s protective barrier. By enabling localized delivery, this method has potential applications in brain tumors, motor system disorders, and other conditions where targeted pharmacological or genetic intervention is beneficial.

“By overcoming the delivery barrier, we can bring effective agents directly to diseased tissue,” Beisteiner notes, underscoring the broad translational implications of blood–brain barrier opening for precision neurology.

Requests to participate in studies

These ultrasound-based treatments are currently offered primarily in a research or specialized clinical context. Because the methods are technically sophisticated, prospective participants require detailed information about risks and benefits, and treatments should be administered by teams with proven expertise in neuroscience and focused ultrasound technologies.

About this neurotech and neurology research news

Source: University of Vienna
Contact: Johannes Angerer – University of Vienna
Image: The image is in the public domain

Original Research: Open access. “Transcranial Ultrasound Innovations Ready for Broad Clinical Application” by Roland Beisteiner and Andres M. Lozano. Advanced Science


Abstract

Transcranial Ultrasound Innovations Ready for Broad Clinical Application

Brain disorders present major challenges in an aging population. Conventional drug treatments are limited and many surgical interventions are invasive. Non-invasive neuromodulation techniques have historically lacked the ability to target deep brain structures with high precision. Transcranial ultrasound represents a versatile, non-surgical platform that now supports three revolutionary clinical strategies: (1) targeted non-invasive surgery, (2) localized delivery of drugs, antibodies, or genes through controlled opening of the blood–brain barrier, and (3) highly focused brain stimulation to improve pathological function. This progress report reviews the current state of the art and summarizes recent patient studies that demonstrate safety, feasibility, and therapeutic promise for these ultrasound-based approaches.