First US Clinical Trial for Alzheimer’s Disease Underway


First Alzheimer’s Disease Patient Treated in a US Clinical Trial

WVU researchers pose with their first
Alzheimer's patient, Judi

Researchers at the West Virginia University (WVU) Rockefeller Neuroscience Institute have treated the first patient in a new, groundbreaking clinical trial utilizing focused ultrasound to open the blood-brain barrier (BBB) in patients with early Alzheimer’s disease. 

Led by neurosurgeon Ali Rezai, MD, the trial uses Insightec’s Exablate Neuro device to temporarily and reversibly open the BBB in regions of the brain affected by Alzheimer’s disease. Researchers will evaluate whether focused ultrasound reduces the debilitating plaques and cognitive decline that are the hallmarks of the disease.   

Alzheimer's disease is the most common neurodegenerative condition, and medical treatments to date have been only modestly effective at slowing cognitive decline and can only treat the symptoms. Researchers believe this is due in part to the BBB – a protective layer of cells that lines the blood vessels of the brain and prevents harmful substances, such as toxins and infections, from entering the brain. Unfortunately, it also prevents adequate amounts of some medications from reaching diseased brain targets. 

In July, results of a similar trial from a team at Sunnybrook Health Sciences Centre in Toronto were published in Nature Communications. That study demonstrated the feasibility and preliminary safety of focally, reversibly, and repeatedly opening the BBB in six patients. 

The first patient in the WVU Rockefeller Neuroscience Institute trial is Judi, a 61-year-old nurse who had to stop working due to disease-induced short-term memory loss. Preliminary results demonstrate that the BBB was safely and successfully opened. After the procedure, Judi was in good spirits and went home.

“I am hopeful that focused ultrasound opening of the blood-brain barrier will prove to be a valuable treatment option for Judi and other patients with early Alzheimer’s who are confronting the enormous challenges associated with the disease on a daily basis,” said Dr. Rezai.

Foundation Chairman Neal F. Kassell, MD, added, “We applaud the team at West Virginia University for their work to advance focused ultrasound therapy for these patients. This is the first trial of its kind in the US, and the pioneering work of Dr. Rezai and his team will expand the body of knowledge that exists for brain applications of focused ultrasound therapy and pave the way for US patients to gain access to treatment.”

“Insightec is honored to collaborate with prominent research teams to advance innovative treatments for patients with Alzheimer’s disease,” said Maurice R. Ferré, MD, chief executive officer and chairman of the Board of Insightec. “The opening of the blood-brain barrier with our focused ultrasound technology is a new frontier that holds enormous potential for treating challenging diseases in the brain.”

In all, there will be 10 patients treated in this pilot trial. Those who are interested in participating at West Virginia University should contact, Anne Bolyard at (304) 293-6318 or

Read the WVU Release >