WVU Rockefeller Neuroscience Institute first in the world to open hippocampal blood brain barrier in Alzheimer’s patients

MORGANTOWN, W.Va. – The West Virginia University Rockefeller Neuroscience Institute today announced a new study published in partnership with Weill Cornell Medical Center that demonstrates the successful opening of the blood brain barrier in the hippocampus and entorhinal cortex using focused ultrasound to treat six patients with early onset Alzheimer’s disease. 

This first-in-the-world study has been published in the Proceedings of the National Academy of Sciences journal. The effort is part of a Phase II clinical trial, sponsored by INSIGHTEC, which developed the technology and manufactures the focused ultrasound device, Exablate Neuro.

“The blood brain barrier has long presented a challenge in treating the most pressing neurological disorders,” Ali Rezai, M.D., executive chair of the WVU Rockefeller Neuroscience Institute, said. “The ability to non-invasively 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.”

The blood-brain barrier (BBB) separates the bloodstream from the brain tissue and limits potentially effective medicines, immunotherapy, gene therapy and other therapeutics of the brain. During this study, doctors injected microscopic bubbles into the patient’s bloodstream, and exposed the bubbles to focused ultrasound from a treatment helmet attached to the MRI, temporarily causing the blood brain barrier to open. Researchers targeted a deep part of the brain, the hippocampus and entorhinal cortex, which are involved in memory processing and are affected early on by Alzheimer’s disease. 

“By working with leading clinicians, researchers, and hospitals, we are gaining tremendous  insight and experience on how our MR-guided focused ultrasound platform can help to treat devastating diseases like Alzheimer’s,” Maurice R. Ferre, M.D., chairman and CEO of INSIGHTEC, said.

This study reported on four subjects treated at the Rockefeller Neuroscience Institute and two at Weill Cornell Medical Center. All patients had early onset Alzheimer’s disease. The six participants safely underwent a total of 17 focused ultrasound treatments with immediate opening of the blood brain barrier and closure within 24 hours with no adverse events. The safe and reversible opening of the BBB provides an opportunity in the future to introduce therapies that would not normally reach the brain, such as medication, gene therapy, and immunotherapy, or to clear out pathological components, such as amyloid plaques in patients with Alzheimer’s. 

“The rapid evolution of focused ultrasound technology from non-invasive treatments for tremor, which was only introduced a few years ago, is astounding,” Michael Kaplitt, M.D., Ph.D., professor of Neurological Surgery at Weill Cornell Medicine, said. “Our demonstration that we can safely and precisely open the blood-brain barrier over most of an important structure in the brain suggests that we may be able to deliver restorative therapies for complex brain disorders without invasive surgery as an outpatient procedure. Given recent events, the potential to treat major brain disease without using inpatient hospital resources could not only be preferable for patients but could provide important options when circumstances limit access to invasive surgery.” 

In late 2018, the Rockefeller Neuroscience Institute launched the first in the U.S. trial using focused ultrasound to treat a patient with early stage Alzheimer’s disease. This first patient was Judi Polak, a nurse who had to stop working due to disease-induced short-term memory loss. Today’s study is a continuation of this effort and validates the novel treatment across five additional patients. 

About the WVU Rockefeller Neuroscience Institute

We are improving lives by pioneering advances in brain health. With the latest technologies, an ecosystem of partners, and a truly integrated approach, we are making tangible progress. Our goal is to combat public health challenges ranging from addiction to Alzheimer’s, benefiting people in West Virginia, neighboring states, and beyond. Learn more about the RNI’s first-in-the-world clinical trials and the top caliber experts joining us in our mission. For more information, please visit WVUMedicine.org/RNI.