New Alzheimer’s Clinical Trial Results


WVU team and Judi captionWest Virginia University’s Rockefeller Neuroscience Institute partnered with Weill Cornell Medicine to publish initial results from their clinical trials using focused ultrasound to open the blood-brain barrier in patients with early-onset Alzheimer’s disease.

In the first six patients of the ongoing, multicenter, phase II trial, successful opening of the blood-brain barrier was demonstrated in two regions of the brain: the hippocampus and the entorhinal cortex. These results confirm previous studies that have demonstrated that the BBB can be safely, reversibly, and repeatedly opened in humans using a combination of focused ultrasound and microbubbles. The study is using Insightec’s ExAblate Neuro device.

“The results of this trial further confirm the feasibility and safety of opening the blood brain barrier in patients with Alzheimer’s disease,” said Foundation Chairman Neal F. Kassell, MD. “This study represents an important step in the path that will potentially lead to a new approach for treating certain patients who are suffering with this devastating disease.”

According to the study, the six subjects underwent 17 focused ultrasound treatments. No adverse events were reported, and cognitive and neurological testing showed no clinical changes within the follow-up period of up to 15 months.

“The blood brain barrier has long presented a challenge in treating the most pressing neurological disorders,” said Ali Rezai, MD, Chair of the Rockefeller Neuroscience Institute. “The ability to noninvasively and reversibly open the blood-brain barrier in deep brain areas such as the hippocampus offers a new potential in developing treatments for Alzheimer’s disease.”

Looking ahead, the team believes that “focal BBB opening will allow targeted delivery of therapeutics to meaningful volumes of essential brain structures in Alzheimer’s disease and other neurological conditions.”

See “Noninvasive hippocampal blood−brain barrier opening in Alzheimer’s disease with focused ultrasound” in the Proceedings of the National Academy of Sciences (PNAS) >

See the WVU Medicine Press Release >