Repositioning Fenofibrate for the Treatment of Neurodegenerative Disease

Categories: “Neurological Disorders

Reference: #2013-062

OTC Contact: Ruchika Nijhara, PhD, MBA, CLP ( Directory Information | Send a Message )


Currently there is no cure for most neurodegenerative diseases including Parkinson’s disease (PD) and Alzheimer’s disease (AD). Studies show that proliferator-activated receptor gamma co-activator-1 alpha (PGC-1alpha) expression levels decreases in PD and AD, while enchanced PGC-1alpha levels protect neural cells from oxidative stress or alpha-synuclein-mediated neuronal death. Thus, PGC-1alpha 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-1alpha to provide protection against neurodegeneration. Specifically, the invention describes a new use of fenofibrate, an FDA approved drug for reducing cholesterol and triglycerides in the blood, to induce PGC-1alpha 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 fenofibrate in vivo, fenofibrate offers great therapeutic potential for the treatment of neurodegenerative diseases.   


Potential new uses of existing therapeutics for the treatment of neurodegenerative disorders including PD, AD, and other neurodegenerative diseases.


Stage of Development

In vitro and in vivo studies promising data on fenofibrate-mediated upregulation of PGC-1a in CNS cells, as well as neuroprotection and anti-inflammation effects at the cellular level in the disease models. Further studies to ascertain behavioral improvements in PD and AD in animal models are on-going.

Patent Status

Patent application pending


Mihael Polymeropoulos; Howard Federoff; Xiaomin Su