Cleveland Clinic launches largest ever clinical study of neurological disorders

Updated: 10:03 a.m., Wednesday, Jan. 19, 2022

The Cleveland Clinic has launched the largest ever clinical study to better understand why millions of people – about one in every six -- around the globe suffer from neurological disorders, including Alzheimer’s disease, Parkinson’s disease, multiple sclerosis, stroke or epilepsy. 

The clinic on Wednesday announced the longitudinal multi-year studythat will collect data from 200,000 neurologically healthy individuals over the course of 20 years to identify brain disease biomarkers and targets for preventing and curing neurological disorders. 

 “What we are hypothesizing here is … there should be some changes - in the blood, in the stool, in the brain, in our ability to think, to walk, to talk – that may give us, if we follow [these] various factors over time, that we may be able to identify something that will change,” said Dr. Imad Najm, principal investigator of the study and director of the Clinic’s epilepsy center. “This will give us the ability to target it for pre-disease diagnosis and hopefully target it for developing new treatment approaches to stop the disease before it happens.” 

Researchers hope to discover causes of neurological disorders and what happens before symptoms become obvious - a time known as the "silent phase," said Dr. Andre Machado, one of the leaders of the study, in a news release.

“Our hope is to change the course of neurodegeneration, with the long-term goal of curing diseases in their earliest stages, years before symptoms are even seen," Machado said.

The study, funded in part by philanthropic contributions of all sizes, launched at the hospital's main campus in Cleveland but will expand to additional Cleveland Clinic sites over time, according to the release. 

In the first five years of the study, researchers will enroll and thoroughly examine 10,000 volunteers, the release said. This group will include adults 50 years and older with no known neurological disorder and neurologically healthy adults age 20 and up who have a first-degree relative with multiple sclerosis. 

The probability that someone will develop a neurological disease increases over time, Najm said. Individuals age 50 to 55 have a one percent chance of developing one, while the risk increases to 14 percent after age 75.

Because of this, the researchers hypothesize some of the older participants may develop a brain disease within the first five years of the study, and investigators can start looking into treatments and possible cures in the following years, Najm added. 

“We think in the first five years … we’ll have some results, which hopefully will pinpoint a target or two for us to build on,” he said, “to stop the disease after we identify the risk factor and the cause of the disease. We don’t think we’re going to cure all diseases in five years, but we think, hopefully, we’ll make a dent for the better understanding of the silent phase, before these crippling disorders happen.” 

You do not need to be a current Cleveland Clinic patient to participate in the study. If a participant is diagnosed with a brain disease at some point during the study, they will either be referred to their primary care physician for treatment, or can opt to be treated at Cleveland Clinic, Najm said. 

Participants will be assessed yearly, undergoing a neurological examination, bloodwork, eye retina scans, brain MRIs, EEG and sleep studies and other cognitive function tests, the clinic said. 

Researchers will collect data points from these assessments to look for trend lines that capture genetic risk factors and invisible molecular, structural, neurophysiological and cognitive changes in the brain over time, according to the release. These “disease fingerprints” may help researchers guide diagnostic and preventative medicine.

Modern medicine has learned how to manage some symptoms of brain disorders, the release said. But doctors continue to struggle to predict who will become sick and how to stop the progression of – let alone cure – these diseases once they are diagnosed.

The study’s leaders hope their research will change that.

“This research will help understand the mechanisms of brain diseases and lead to the design of preventive treatment for neurological diseases. This is precision medicine in its best form,” Najm said. “We’re building a foundation to screen one person at a time – potentially with something as routine as a blood test – to diagnose brain disease on the spot and prevent it from happening altogether."