New clinical trial underway for Alzheimer's patients

Baptist Health Miami Neuroscience Institute and Florida International University’s Herbert Wertheim College of Medicine are collaborating to offer a new clinical trial for patients with Alzheimer’s disease. The study uses low-intensity focused ultrasound to disrupt brain changes that lead to the neuron damage that causes memory and cognition problems in patients.

The investigational study, called ExAblate Blood-Brain Barrier (BBB) Disruption for the Treatment of Alzheimer’s Disease, could revolutionize care for those suffering from Alzheimer’s, said co-principal investigator Michael McDermott, M.D., neurosurgeon and chief medical executive of Miami Neuroscience Institute and professor and chief of the Division of Neuroscience at the Herbert Wertheim College of Medicine.

“The technology is very exciting, especially because there is no cure for Alzheimer’s and only a few drug treatments that temporarily treat symptoms,” Dr. McDermott said. “It’s non-invasive for patients and we hope it will lead to cognitive improvement.” 

The FDA-approved clinical trial, currently enrolling patients, is part of Florida’s Brain State initiative, which funds and brings together hospitals, state universities and institutions in Florida. Alzheimer’s disease is the sixth leading cause of death for those age 65 and above in the U.S., according to the Alzheimer’s Association. Florida has the second highest incidence of Alzheimer’s with nearly 600,000 cases.

The high intensity ultrasound technology, developed by Insightec, has already proven to be a game changer for patients who cannot perform common tasks such as holding a cup of water without spilling, shaving safely or writing legibly due to essential tremor. For essential tremor, high-intensity focused ultrasound (HiFU) waves are targeted to hit the area of abnormal circuitry in the brain. In one sitting, patients see immediate improvement.

Geriatric psychiatrist and Alzheimer’s study co-principal investigator Patricia Junquera, M.D., hopes to see similar results with the low-intensity ultrasound. “We expect to see improvement days after the procedure,” said the associate professor and vice chair of clinical services for the Department of Psychiatry and Behavioral Health at FIU’s College of Medicine.  “Any improvement we see is going to be huge because patients with Alzheimer’s typically cannot make any new memories or manage functions of daily living.”

To determine if they are eligible for the study, patients must first be referred to Dr. Junquera by their primary care physician, neurologist or psychiatrist. An evaluation at FIU will be performed to determine that a patient’s dementia is caused by Alzheimer’s and not due to other issues.

The evaluation includes a battery of psychological tests and can take several hours. A caretaker or family member must also be present.

Next, the patient is seen at Baptist Hospital, where Dr. McDermott performs additional medical and imaging tests.

If the patient meets study inclusion criteria, study participation begins with the affixing of a stereotactic frame to the scalp under local anesthesia. Using MRI guidance, the ultrasound waves are directed to pre-determined areas of the brain. Patients are observed for several hours and are also seen the next day. The study involves three treatments, two weeks apart. After each session and at checkpoints along the way, patients will repeat imaging and psychological testing. Dr. Junquera will continue to follow the patients for 5 years after the final treatment.

In addition to Baptist Health and FIU, other organizations participating in the clinical trial are Florida Atlantic University, Delray Medical Center, University of Florida, Broward Health, Weill Cornell Medical College, Focused Ultrasound Foundation, and Insightec. The research builds upon a smaller earlier study by Weill Cornell Medicine, The Ohio State University – Wexner Medical Center and West Virginia University Rockefeller Neuroscience Center. The Florida Legislature has approved funding for the study.