A new treatment using focused ultrasound to treat early-stage Alzheimer’s disease was successfully administered to a patient. The treatment, which involves incisionless surgery and no pharmaceuticals, is part of a worldwide Phase 2 trial.
The trial (NCT03671889) is underway and recruiting up to 10 participants — men or women 50-85 years old with probable Alzheimer’s disease. It will be conducted at up to four sites in the United States.
This clinical study is examining whether focused ultrasound diminishes the toxic protein aggregates and cognitive dysfunction that are hallmarks of Alzheimer’s disease. This type of non-invasive treatment involves the application of acoustic energy at low frequencies into distinct targets to induce blood-brain barrier (BBB) disruption in the regions of the brain affected by Alzheimer’s disease, such as the hippocampus.
The BBB separates the brain from the bloodstream for protective reasons; however, the BBB limits the immune system, for immunotherapy, and many potentially effective pharmaceutical treatments from reaching diseased brain areas.
During the summer of 2017, researchers at Sunnybrook Health Sciences Centre in Toronto reported the results of a Phase 1 clinical trial (NCT02986932) showing that they could safely open and reseal the BBB in six patients with early-stage Alzheimer’s. The current Phase 2 trial is the next phase to test the effectiveness of focused ultrasound as a treatment for Alzheimer’s.
The procedure was performed by a team from the WVU Rockefeller Neuroscience Institute led by neurosurgeon Ali Rezai, M.D., in collaboration with INSIGHTEC, an Israeli medical technology company. INSIGHTEC was approved earlier in the year by the U.S. Food and Drug Administration (FDA) to begin the Phase 2 trial of the procedure and selected the WVU Rockefeller Neuroscience Institute as the first site in the United States for the trial.
“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,” Maurice R. Ferré, M.D., chief executive officer and chairman of the board of INSIGHTEC, said in a press release.
The procedure begins by injecting tiny bubbles into the bloodstream of a patient. When exposed to focused ultrasound, the bubbles cause the BBB to open briefly at the targeted site in the brain.
For this study, the team targeted the hippocampus and the brain’s memory and cognitive centers that are affected by amyloid-beta plaques found in Alzheimer’s patients.
A 61-year-old nurse with early-stage Alzheimer’s who had to stop working because of disease-induced short-term memory loss was the first patient to receive the treatment. Over the course of three hours, she underwent MRI scans and focused ultrasound of the hippocampus. The results show that the BBB was safely and successfully opened.
The possible benefits of these treatments will take a few years to evaluate comprehensively.
“I am hopeful that focused ultrasound opening of the blood-brain barrier will prove to be a valuable treatment option for … patients with early Alzheimer’s who are confronting the enormous challenges associated with the disease on a daily basis,” Rezai said.
For more information on how to participate in the trial at WVU, call Barbara Harring at 304-293-9638.