Phase 1 Trial Begins Evaluating Safety of Focused Ultrasound in Alzheimer’s Patients

Insightec’s new platform, Exablate Neuro, is to be a part of the first-ever clinical trial (NCT02986932) evaluating the safety and feasibility of opening the blood-brain barrier (BBB) in Alzheimer’s disease patients at Sunnybrook Health Sciences Centre in Toronto, Canada.

Exablate Neuro uses a technique called focused ultrasound, a noninvasive therapeutic technology. It applies ultrasonic energy to target tissues that are located deep within the brain without the use of incisions or radiation.

Multiple beams are projected through the brain with no individual effect. Once they converge on a focal point, the ultrasound then works towards healing the tissue. This technology is currently being explored for the treatment of a variety of diseases, including essential tremor, Parkinson’s disease, and Alzheimer’s.

Six Alzheimer’s patients, ages 50-85, will be enrolled in this Phase 1 trial. The procedure involves two rounds of noninvasive focused ultrasound. The first stage involves focused ultrasound with Exablate Neuro to a small area in the right frontal lobe.

The second stage involves a similar procedure, where the focused ultrasound will target a larger area of the right frontal lob. Images obtained from each procedure will allow researchers to determine whether the blood-brain barrier was opened.

Previous work conducted in animal models demonstrated that opening the blood-brain barrier using focused ultrasound can allow immune cells to cross into the brain. Once that happens, these cells can work toward clearing away the amyloid-beta plaques that are characteristic of Alzheimer’s, which can then hopefully lead to improvements in behavioral and cognitive issues. If this technology proves to be successful in patients with early Alzheimer’s, it could potentially stop the progression of the disease.

The opening of the blood-brain barrier means potentially effective Alzheimer’s disease therapies would be able to breach the barrier, which is currently a major challenge in treatment.

“This effort is a small but critical step in what could potentially be a game-changing treatment by enhancing drug delivery to the areas of the brain where they are needed, and in much higher concentrations than can be achieved through intravenous or oral administration,” Neal Kassell, MD, chairman of the Focused Ultrasound Foundation, said in a press release. The foundation is funding the trial.

Eyal Zadicario, general manager at Insightec Israel, whose Exablate system is being used in the trial, said the study is a “significant milestone in the development of our MR-guided focused ultrasound technology.”

“Exablate Neuro was designed as a platform to support a wide range of transcranial applications and expand innovation in the neuroscience field,” Zadicario said. “In the current study, Exablate Neuro is used in a new approach — to tackle neurodegenerative diseases by opening the BBB. This is just a first step, and we will continue to push the technology to make significant impact where it matters most — to patients.”