MORGANTOWN, W.Va. – A groundbreaking study led by the West Virginia University Rockefeller Neuroscience Institute (RNI) demonstrating the safety and feasibility of using focused ultrasound technology to open the blood-brain barrier (BBB) in patients with Alzheimer’s disease has been published in the peer-reviewed Journal of Neurosurgery.  

The RNI team’s research found that low-intensity focused ultrasound (LIFU) can non-invasively, safely, and reproducibly open the blood-brain barrier in multiple brain regions with beta amyloid deposits in Alzheimer’s disease. The published study is the largest cohort of patients with Alzheimer’s treated with focused ultrasound. The study’s results showed no worsening of cognitive function as compared to a matched Alzheimer’s control group and revealed mild beta amyloid reduction in 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,” Ali Rezai, M.D., executive chair of the RNI, said.

“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.”

Alzheimer’s disease is the most common form of dementia, causing progressive memory and cognitive decline, and its incidence is rising worldwide. There is no sustained effective treatment or cure for it, and treatment approaches involve medications that temporarily slow the worsening of symptoms for a short time.

The study, sponsored by INSIGHTEC, reports on 10 participants aged 55 to 73 from the WVU Rockefeller Neuroscience Institute and Weil Cornell Medical College, each with mild Alzheimer’s disease and undergoing noninvasive focused ultrasound. Opening the blood-brain barrier allowed access to areas of the brain critical to learning and retaining memories where beta amyloid plaques were present. The targeted regions included the hippocampus, the entorhinal cortex, and the frontal and parietal lobes. All patients showed blood-brain barrier closure within 24-to-48 hours, and none of the patients showed serious adverse events related to the procedure.  

“Insightec is committed to collaborating with leading researchers to advance the use of acoustic therapy in the brain,” Maurice R. Ferré, M.D., Insightec CEO and chairman of the board, said. “Use of our low-intensity focused ultrasound technology to open the blood-brain barrier for the clearance of amyloid plaques is an exciting advance that holds promise to become a paradigm shift in the treatment of patients with Alzheimer’s disease.”