Ali Rezai, M.D., and team review the precise area of the brain where the blood-brain barrier will temporarily open, allowing drug therapy to enter.

West Virginia University Rockefeller Neuroscience Institute expands groundbreaking Alzheimer’s research in latest trial

MORGANTOWN, W.Va. – The West Virginia University Rockefeller Neuroscience Institute (RNI) has launched a first-in-the-world clinical trial pairing focused ultrasound treatment with Alzheimer’s antibody drug therapy to a targeted area of the brain, unlocking new potential for the treatment of the disease. 

The first participant, a 77-year-old man from West Virginia, received the combination of Aduhelm (generic: aducanumab), a monoclonal antibody treatment that targets beta amyloid plaques as part of the FDA-approved trial. With the antibody in the patient’s bloodstream, researchers aimed ultrasound waves to specific brain regions with beta amyloid to temporarily open the blood-brain barrier to facilitate drug delivery. The procedure was the first of a series of treatments the patient will undergo over six months.

While Aduhelm was FDA approved for the treatment of Alzheimer’s last year, its effects may be limited by ability to cross the protective blood-brain barrier. By opening this barrier and allowing the drug to enter the brain in key areas where amyloid plaques are present, researchers hope to potentially accelerate the plaque-clearing process while also delivering smaller doses of the drug, limiting potential side effects. 

In 2018, the RNI became the first in the U.S. to use focused ultrasound to treat a patient with early-stage Alzheimer’s disease. The RNI team has since expanded treatment to multiple brain locations in that ongoing clinical trial, a partnership with INSIGHTEC. The team’s peer-reviewed studies have been published in the Proceedings of the National Academy of SciencesFrontiers in Human Neuroscience, and Radiology.

“After years of successfully using focused ultrasound to safely and locally open the blood-brain barrier, we’ve now taken a critical step forward in pairing this capability with therapeutic treatment,” Ali Rezai, M.D., executive chair of the WVU Rockefeller Neuroscience Institute, said. “We thank our patient and his family for being pioneers for this first-in-the-world study, which we hope leads to long-overdue relief for those struggling with Alzheimer’s.”

The RNI team again partnered with INSIGHTEC, which developed the technology and manufactures the focused ultrasound device, Exablate Neuro.

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