Repositioning Fenofibrate for the Treatment of Neurodegenerative Disease

OVERVIEW

Methods of treating or preventing neurodegenerative diseases associated with proliferator-activated receptor gamma co-activator-1α (PGC-1α) deficiency and mitochondrial disorder. The new use of fenofibrate, an FDA-approved drug for reducing cholesterol and triglycerides in the blood, leads to inducing PGC-1alpha expression in neuronal and glial cells.

BACKGROUND

Currently, there is no cure for most neurodegenerative diseases, including Parkinson’s disease (PD) and Alzheimer’s disease (AD). Studies show that PGC-1α expression levels decrease in PD and AD, while enhanced PGC-1α levels protect neural cells from oxidative stress or alpha-synuclein-mediated neuronal death. Thus, PGC-1α is a promising therapeutic target within the central nervous system for the treatment of these neurodegenerative diseases. The present invention describes a novel approach to pharmacologically upregulate PGC-1α, providing protection against neurodegeneration. Specifically, the invention describes a new use of fenofibrate to induce PGC-1α expression in neuronal and glial cells. Fenofibrate exerts both neuroprotective and anti-inflammatory effects in neuronal and glial cells. Given the demonstrated safety profile of fenofibratein vivo, fenofibrate offers great therapeutic potential for the treatment of neurodegenerative diseases.

Benefit

• The first proposal was to use fenofibrate for an early stage of Parkinson’s disease.
• Demonstrated safety profile.
• Employs existing FDA-approved drugs, and this potentially expedites regulatory approval.
• Provides an anti-inflammatory effect and increases neural cell viability.

Market Application

• Proven efficacy in the prevention and treatment of various neurodegenerative conditions, such as Parkinson's Disease, Parkinson-plus syndrome, familial dementia, Alzheimer's Disease, Huntington's Disease, multiple sclerosis, and more.
• Method for screening for neuroprotective agents.

Publications

• US Patent Application No. 15/022,654. Corresponding foreign patents were granted in France, Germany, Italy, Spain, the United Kingdom, Canada, South Korea, and Japan and are pending in China.