Cleveland Clinic Participating in Clinical Trials to Test Treatments for Devastating, But Little-Known, Brain Disease

First U.S. clinical trials test drugs to treat Dementia with Lewy Bodies

Cleveland Clinic will participate in a series of studies designed to find treatments for a little-known but surprisingly common brain disease, Dementia with Lewy Bodies.

The progressive neurological disorder is the second most common form of neurodegenerative dementia, after the better-known Alzheimer’s disease. However, there are neither drugs that target the disease nor treatments that offer meaningful hope for a cure.

Cleveland Clinic is now enrolling patients in the first U.S. clinical trials designed to test two investigational drugs to treat Dementia with Lewy Bodies (DLB), which afflicts more than one million Americans. Researchers hope for results next year.

The studies, sponsored by drugmaker Axovant Sciences, involve about two dozen medical centers around the world.

“It’s an exciting opportunity,” said  Babak Tousi, M.D., head of the Clinical Trials Program at the Cleveland Clinic Lou Ruvo Center for Brain Health. “The hope would be that we might finally have some treatments, some approved medications, to offer these patients and their families.”

Dr. Tousi is the principal investigator for the Cleveland site. The six-month study will enroll about 240 patients nationally to test the safety and effectiveness of RVT-101, a tablet that researchers think can restore cognitive function, or thinking skills, in DLB patients.

A second clinical trial will be led in Cleveland by James Leverenz, M.D, the director of the Cleveland Clinic Lou Ruvo Center for Brain Health and chair of the Scientific Advisory Council of the national Lewy Body Dementia Association.  The five-month study will investigate the safety and effectiveness of nelotanserin, a medication that researchers think can help people suffering from hallucinations and sleep disturbances.

Both trials are currently recruiting patients with the disease who are between the ages of 50 and 85. Interested participants should call the Lou Ruvo Center for Brain Health at 216-445-9009.

Dementia with Lewy Bodies is caused by the buildup of abnormal protein deposits, called “Lewy Bodies,” in brain cells. It was discovered in the early 1900s by scientist Frederick H. Lewy and can only be confirmed by a post-mortem brain autopsy.

According to Dr. Tousi, DLB may account for more than 15 percent of all cases of dementia. It’s often confused with Alzheimer’s and Parkinson’s diseases, which share causes and symptoms, and DLB patients are often misdiagnosed.

People with Lewy Bodies in their brain often experience rapid decline in their balance, motor skills and memory. They tend to be younger than people with Alzheimer’s and deteriorate more rapidly, often suffering anxiety attacks, hallucinations and nightmares. Many patients might have experienced acting out dreams for years prior to showing difficulty with thinking.

Despite some high-profile patients, Dementia with Lewy Bodies has been eclipsed by more prominent forms of dementia, which means there has been little research into a cure.

Lacking tested remedies, DLB patients often are treated with “off label” drugs designed for other diseases, sometimes with harmful consequences, Dr. Tousi said.

RVT-101 has been shown to raise levels of acetylcholine, a vital chemical in the brain that affects memory and behavior. Deficits in acetylcholine are a prominent feature of DLB.

Meanwhile, nelotanserin, a drug that has shown promise calming neuropsychiatric disturbances, will be tested for its ability to manage the visual hallucinations brought on by DLB.

For more information about the clinical trials, patients can visit:



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Cleveland Clinic is a nonprofit multispecialty academic medical center that integrates clinical and hospital care with research and education. Located in Cleveland, Ohio, it was founded in 1921 by four renowned physicians with a vision of providing outstanding patient care based upon the principles of cooperation, compassion and innovation. Cleveland Clinic has pioneered many medical breakthroughs, including coronary artery bypass surgery and the first face transplant in the United States. U.S.News & World Report consistently names Cleveland Clinic as one of the nation’s best hospitals in its annual “America’s Best Hospitals” survey. More than 3,000 full-time salaried physicians and researchers and 11,000 nurses represent 120 medical specialties and subspecialties. The Cleveland Clinic health system includes a main campus near downtown Cleveland, eight community hospitals, more than 90 northern Ohio outpatient locations, including 18 full-service family health centers, Cleveland Clinic Florida, the Lou Ruvo Center for Brain Health in Las Vegas, Cleveland Clinic Canada, and Cleveland Clinic Abu Dhabi. In 2014, there were 5.9 million outpatient visits throughout the Cleveland Clinic health system and 152,500 hospital admissions. Patients came for treatment from every state and 147 countries. Visit us at  Follow us at

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