Alzheimer’s disease: In a groundbreaking development, researchers at the West Virginia University Rockefeller Neuroscience Institute (RNI) have reported promising results from the first human trials of a novel Alzheimer’s therapy. The study, published in The New England Journal of Medicine on January 11, highlights the success of a unique approach that combines focused ultrasound (FUS) with anti-amyloid-beta monoclonal antibody treatments in accelerating the removal of amyloid-beta plaques from the brains of patients with Alzheimer’s disease.
Alzheimer’s disease, a progressive neurodegenerative disorder, is characterized by the abnormal buildup of amyloid-beta proteins in the brain. These proteins clump together, forming plaques that interfere with normal neuronal function and contribute to the cognitive decline seen in Alzheimer’s patients.
Anti-amyloid-beta monoclonal antibody treatments, such as aducanumab and lecanemab, have demonstrated efficacy in clearing these plaques and slowing disease progression. However, the blood-brain barrier (BBB), a protective barrier designed to prevent harmful substances from reaching the brain, has limited the effectiveness of these drugs. More than 98% of drugs are typically blocked by the BBB, necessitating higher doses and more frequent therapies for patients.
In this innovative study, scientists utilized a focused ultrasound (FUS) system to temporarily open the blood-brain barrier, allowing antibodies to penetrate areas of the brain with high amyloid-beta plaque concentrations. After six months of treatment with anti-amyloid-beta antibodies, participants exhibited an average of 32% more reduction in amyloid-beta plaques in areas where the BBB was opened compared to areas where the drug was used without ultrasound.
Dr. Ali Rezai, the lead researcher and director of the Rockefeller Neuroscience Institute at WVU, emphasized the significance of the findings. “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,” said Dr. Rezai. He added that non-invasive focused ultrasound is an outpatient procedure allowing targeted delivery of therapeutics to the brain, potentially enhancing the benefits of antibody treatment in Alzheimer’s disease.
The study included three participants, aged between 59 and 77, all diagnosed with mild Alzheimer’s disease. Each participant received six monthly infusions of the aducanumab antibody, with focused ultrasound used to open the blood-brain barrier at sites of high plaque buildup. Importantly, the researchers confirmed the temporary nature of the BBB opening through MRI scans, assuaging concerns about potential side effects such as brain swelling and hemorrhage.
The reductions in amyloid plaques were verified through PET scans, marking a crucial first step toward larger studies. In subsequent phases of the clinical trial, the ultrasound therapy will be combined with lecanemab, another anti-beta-amyloid antibody.
Dr. James Galvin, director of the Comprehensive Center for Brain Health at UHealth, the University of Miami Health System, who was not involved in the research, emphasized the significance of the study. “A study like this is important because it demonstrates that there may be safe ways to increase drug delivery to the brain without any serious adverse effects,” said Dr. Galvin.
As the research progresses, larger studies will enable researchers to evaluate more patients and larger areas of the brain, offering hope for an innovative and effective treatment approach for Alzheimer’s disease.
Alzheimer’s disease:
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