The results of the first ever clinical trial of focused ultrasound to open the blood-brain barrier (BBB) in patients with Alzheimer’s disease were published today in Nature Communications and also presented at the Alzheimer’s Association International Conference (AAIC) in Chicago, Illinois. The pilot trial demonstrated the feasibility and preliminary safety of focally, reversibly, and repetitively opening the BBB.

“This groundbreaking trial is the first small, but critically important, step in a process that could potentially lead to a novel approach to delivering drugs to the brain to treat Alzheimer's disease,” said Neal F. Kassell, MD, founder and chairman of the Focused Ultrasound Foundation. “This study widens the horizon of possibility for focused ultrasound to become a treatment option for many neurological disorders by demonstrating that the technology can, in fact, be used to open the blood-brain barrier to allow drugs in higher concentrations, and those that may otherwise be excluded, to reach the brain.”

The study, “Blood-Brain Barrier Opening in Alzheimer’s Disease Using MR-guided Focused Ultrasound,” was conducted at Sunnybrook Health Sciences Centre in Toronto under the direction of Nir Lipsman, MD, PhD, Director of the Harquail Centre for Neuromodulation, and used Insightec’s Exablate Neuro device. The Focused Ultrasound Foundation funded and facilitated the organization of the trial.

Six Alzheimer’s patients, ages 50-85, underwent two noninvasive focused ultrasound procedures. For the first stage, focused ultrasound was applied in a small area of the brain to temporarily open the BBB. In the second stage, approximately 1 month later, a similar procedure to the same patient targeted a larger area of the brain. It is important to note that this trial was a feasibility and safety study to see if focused ultrasound can be used to repeatedly open the BBB temporarily in these patients, and no drugs were administered in the study.

Dr. Lipsman is presenting data from this study today at AAIC and taking part in a panel discussion regarding the application of focused ultrasound to treat or manage Alzheimer’s disease. Other panelists include Kullervo Hynynen, PhD (Sunnybrook Research Institute); Isabelle Aubert, PhD (Sunnybrook Research Institute); Cynthia Lemere, PhD (Brigham & Women’s Hospital/Harvard University); and Jürgen Götz, PhD (Queensland Brain Institute).

“By successfully opening the BBB safely and reversibly in patients with early to moderate Alzheimer’s disease, we can support the continued investigation of focused ultrasound as a potential novel treatment, and further study the delivery of therapies that otherwise cannot access the brain,” said Dr. Lipsman. “While it is still early in development, focused ultrasound may in the future provide a noninvasive, effective way of delivering large molecules, such as antibodies, directly to the brain to help patients with Alzheimer’s.”

The next phase will investigate the safety and efficacy of using focused ultrasound to breach the BBB in a larger group of patients with Alzheimer’s in a phase 2a trial beginning this fall.

“This research using Insightec’s Exablate Neuro device is truly groundbreaking as it explores a new application for focused ultrasound,” says Maurice R. Ferré, MD, Insightec’s CEO and Chairman of the Board of Directors. “The disruption of the BBB has the potential to break down the barriers to treatment options in the brain with the promise to help millions of patients around the globe.”

—Newswise