Discover How AI Technology is Revealing Genetic Insights and New Treatment Opportunities for Parkinson’s Disease

Researchers at the Cleveland Clinic Genome Center are making strides in understanding Parkinson’s disease using advanced AI models. They focused on identifying genetic factors that contribute to the disease’s progression and suggested existing FDA-approved drugs that might be adapted for treatment.

The study, published in npj Parkinson’s Disease, employs a method called “systems biology.” This technique utilizes AI to merge various types of data—genetic, proteomic, pharmaceutical, and patient records—to uncover patterns that might not be apparent when looking at a single source of information.

Feixiong Cheng, PhD, who leads the study, is a recognized expert in systems biology. He has previously developed AI tools aimed at finding new treatments for conditions like Alzheimer’s disease.

“Parkinson’s is the second most common neurodegenerative disorder after dementia. Unfortunately, we currently have no way to stop or slow its progression,” states Lijun Dou, PhD, the study’s first author. “Our focus is on creating new therapies that can actually change the course of the disease.”

Developing treatments that can halt or even reverse Parkinson’s is complex. Researchers are still determining which genetic mutations lead to specific symptoms. Many known mutations are located in non-coding regions of DNA, not in protein-coding genes. While these areas can influence gene function, the exact genes affected in Parkinson’s are still under investigation.

Using their AI model, the team mapped genetic variations linked to Parkinson’s against specialized brain DNA databases. This helped them identify specific brain genes potentially impacted by non-coding mutations. They also analyzed protein datasets to see how these genes might influence brain function. Several risky genes emerged, including SNCA and LRRK2, which have been linked to inflammation in the brain.

“People living with Parkinson’s can’t wait years for new treatments. By repurposing FDA-approved drugs, we can potentially offer them new options much faster.”

Feixiong Cheng, PhD, Director, Cleveland Clinic Genome Center

The team then looked for existing medications that could act on these identified genes. They discovered that patients taking simvastatin, a cholesterol drug, had fewer Parkinson’s diagnoses. This finding suggests simvastatin might have protective effects worth exploring.

Moving forward, Dr. Cheng plans to test simvastatin’s effectiveness against Parkinson’s in the lab, along with other promising medications that emerged from their study.

“Traditional methods to pinpoint genes, proteins, and drugs are time-consuming. Our network-based approach accelerated the process significantly, increasing our chances of finding new solutions,” Dr. Dou explains.

This study received support from the National Institutes of Health (NIH) through grants from the National Institute on Aging (NIA) and the National Institute of Neurological Disorders and Stroke (NINDS).