Fox News recently reported that researchers at West Virginia University’s Rockefeller Neuroscience Institute have unveiled a promising new therapy for Alzheimer’s disease. This approach combines focused ultrasound technology with antibody therapies to accelerate the removal of amyloid-beta plaques from the brain, a key factor in Alzheimer’s progression. The findings, published in The New England Journal of Medicine on January 11, 2024, represent a significant leap forward in the treatment of this neurodegenerative disease.

Dr. Ali Rezai, director of the Rockefeller Neuroscience Institute at WVU, emphasized the impact of this research, stating, “This was a first in human safety and feasibility study in three participants demonstrating that the BBB opening can accelerate clearance of beta amyloid plaques.” 

This breakthrough not only highlights the potential of ultrasound to enhance drug delivery across the blood-brain barrier but also sets the stage for a new era in Alzheimer’s treatment, promising a more effective approach to managing and potentially reversing the disease.

The challenge of amyloid-beta plaques in Alzheimer’s

A key cause in Alzheimer’s disease is the accumulation of amyloid-beta proteins in the brain. These proteins clump together to form plaques, which interfere with neuronal function and contribute significantly to the disease’s cognitive decline. Addressing these plaques has been a primary focus of treatment strategies.

Traditionally, anti-amyloid-beta monoclonal antibody treatments like aducanumab and lecanemab have shown promise in clearing these plaques and slowing the disease’s progression. 

However, a major hurdle in their effectiveness is the blood-brain barrier, a natural defense mechanism designed to protect the brain from harmful substances. Unfortunately, this barrier also restricts most therapeutic drugs from reaching the affected brain areas, limiting the potential impact of these treatments.

The blood-brain barrier’s selective permeability means that, for these drugs to be effective, higher doses and more frequent therapies are often required, posing challenges in treatment efficacy and patient well-being. This limitation underscores the need for innovative approaches to enhance drug delivery across the blood-brain barrier, making the recent advancements in focused ultrasound technology a significant step toward overcoming these obstacles.

This technique, developed by West Virginia University researchers, strategically opens the blood-brain barrier, enabling anti-amyloid therapies to reach their target more effectively. 

Promising results from initial human trials

The initial human trials conducted at the Rockefeller Neuroscience Institute have shown promising results, demonstrating a significant reduction in amyloid-beta plaques. This study involved three participants, aged between 59 and 77, all diagnosed with mild Alzheimer’s disease. Participants underwent treatment combining focused ultrasound (FUS) with antibody therapies, specifically targeting areas of the brain affected by amyloid-beta plaques.

Participants treated with this innovative approach experienced an average of 32% more plaque reduction in areas where the blood-brain barrier was temporarily opened using FUS, compared to areas treated without the use of ultrasound. Importantly, MRI scans conducted post-treatment verified that the BBB’s opening was temporary and safely closed within 24 to 48 hours after the procedure, with no serious adverse effects observed, such as brain swelling or hemorrhage. 

Expert insights and external perspectives

The ultrasound-enhanced drug delivery method has received a lot of attention and optimism from experts across the neuroscience field. Dr. James Galvin, Director of the Comprehensive Center for Brain Health at UHealth, acknowledges the innovation as a potentially safe way to enhance drug delivery to the brain without serious adverse effects. While cautious about the preliminary nature of the research, given its small scale, he expresses eagerness for larger, follow-up studies.

Rebecca M. Edelmayer, PhD, from the Alzheimer’s Association, finds the results “very intriguing” and indicative of a promising avenue for improving Alzheimer’s medications’ effectiveness. 

Dr. Hakonarson, founder of Arctic Therapeutics, offered his perspective on the recent findings: “The focused ultrasound technique represents a pivotal advance in our collective effort to combat Alzheimer’s disease. Its ability to enhance the delivery of therapeutic agents across the blood-brain barrier directly aligns with the innovative pathways we’re exploring with AT-001. This technology could significantly augment the efficacy of treatments by ensuring higher concentrations of critical medications reach their intended targets in the brain. 

For AT-001, which is designed to address the molecular underpinnings of Alzheimer’s, such advancements in drug delivery technology not only bolster our current strategies but also open new avenues for therapeutic interventions. It’s a compelling example of how cross-disciplinary innovations can fuel the next generation of Alzheimer’s care.”


Focused ultrasound technology represents a significant advancement in treating Alzheimer’s, offering a new method to reduce amyloid-beta plaques effectively. This breakthrough, alongside innovations like AT-001 from Arctic Therapeutics, highlights the critical importance of continued targeted research and innovation in Alzheimer’s care.

The path forward demands our support for these pioneering efforts. By investing in research and embracing new technologies, we can move closer to transforming Alzheimer’s treatment. It’s essential to keep pushing the boundaries of what’s possible in medical science to benefit those affected by Alzheimer’s.

To learn more about Arctic Therapeutics and AT-001, visit our website here.