Key Points
- Since 2017, researchers have been using focused ultrasound to temporarily open the blood-brain barrier (BBB) in patients with Alzheimer’s disease.
- In October 2018, a US clinical trial investigating this technique began at West Virginia University.
- An update on the first 10 patients in that study has now been published in the Journal of Neurosurgery.
Since 2017, clinical researchers from around the world have been using focused ultrasound to temporarily open the blood-brain barrier (BBB) in patients with Alzheimer’s disease. In October 2018, the US clinical trial investigating the technique began at West Virginia University Rockefeller Neuroscience Institute (RNI), with additional sites – including Weill Cornell Medicine – later joining the study.
The trial is using Insightec’s Exablate Neuro device to temporarily and reversibly open the BBB in regions of the brain affected by Alzheimer’s disease. Researchers are evaluating whether focused ultrasound is safe in multiple brain regions and whether it can reduce the debilitating plaques and cognitive decline that are the hallmarks of the disease.
Interim data from the first ten participants enrolled in the clinical trial have now been published in the Journal of Neurosurgery, reporting the following findings:
- Successful BBB opening in multiple brain regions with no adverse events
- No worsening of cognitive function compared with control group
- Mild beta amyloid reduction in the areas treated with focused ultrasound
“Our results are encouraging, demonstrating the safety of focused ultrasound in multiple brain locations with the potential for reduction in beta amyloid,” said Ali Rezai, MD, executive chair of the RNI, in a press release. “This study is also a major step forward for the exciting possibility of combining focused ultrasound with targeted delivery of medications or antibodies that normally have limited capability to cross the blood brain barrier from the blood to the brain.”
In 2022, additional clinical sites in Florida and Texas were added to the US trial, which plans to enroll at least 30 participants. Although slowed by the pandemic, updates from the trial have been published over time, include the following reports:
Publications from the US Clinical Trial
October 2018
Milestone: Trial begins, first participant treated
Publication: Press release
April 2020
Milestone/Results: First six participants treated, successful BBB opening in two brain regions
Publication: Proceedings of the National Academy of Sciences (PNAS)
December 2020
Results: First six participants’ β-amyloid plaque levels decreased
Publication: Frontiers in Human Neuroscience
March 2021
Results: Contrast agent enhancement patterns from three trial participants reveal unique downstream pattern
Publication: Radiology
November 2022
Results: Ten participants treated, successful BBB opening in multiple brain regions, no worsening of cognitive function compared with control group, mild beta amyloid reduction in the areas treated with focused ultrasound
Publication: Journal of Neurosurgery
See the Journal of Neurosurgery >
See the Press Release from West Virginia University Rockefeller Neuroscience Institute >
Past Coverage: Worldwide Clinical Trials for Focused Ultrasound for Alzheimer’s
Korean Alzheimer’s Disease Study Results Published December 2021
Radiologists Study Contrast Enhancement Patterns from Alzheimer’s Trial March 2021
Beta-Amyloid Plaque in Alzheimer’s Disease Decreased in Focused Ultrasound Trial Patients December 2020
First Alzheimer’s Disease Patient Enrolled in Clinical Trial at Columbia University December 2020
New Alzheimer’s Clinical Trial Results April 2020
New Alzheimer’s Disease Trial Begins in New York February 2020
AARP Features First Alzheimer’s Patient in US Trial December 2019
PBS NewsHour Highlights Focused Ultrasound for Alzheimer’s Disease October 2019
Ohio State Joins Alzheimer’s Clinical Trial May 2019
New Alzheimer’s Disease Trial Begins in Toronto January 2019
Ali Rezai: Emerging Applications of Focused Ultrasound – Alzheimer’s Disease January 2019
Scientific American Article Highlights Focused Ultrasound for Alzheimer’s November 2018
Judi’s Journey with Alzheimer’s October 2018
First US Clinical Trial for Alzheimer’s Disease Underway October 2018
US FDA Approves Initiation of Focused Ultrasound Trial for Alzheimer’s July 2018
Results of Landmark Focused Ultrasound Trial for Alzheimer’s Published July 2018
First Alzheimer’s Trial with Focused Ultrasound Begins May 2017