The use of Pan-PPAR Agonists for Treatment of Tauopathies and Huntington's disease
- Others
-
Johri A et al. (2012) Pharmacologic activation of mitochondrial biogenesis exerts widespread beneficial effects in a transgenic mouse model of Huntington's disease. Hum Mol Genet. 2012 Mar 1;21(5):1124-37.
- *Abstract
-
- Repurposing of bezafibrate, or novel combinations of selective PPAR agonists
- Treatment of neurological conditions
- Published data on treatment of HD mice
- Unpublished/confidential data on treatment of tauopathy mouse model
Peroxisome Proliferator-Activated Receptors (PPARs) are a group of transcription factors that are targets for marketed drugs to treat diabetes and high cholesterol. There are three PPAR subtypes, α, β and γ.
Neuroscientists have also explored utility of PPAR agonists. PPARγ agonists have been shown to decrease neuron cell loss, inflammation, and extend survival in mouse models of neurodegeneration, including amyotrophic lateral sclerosis and Alzheimer’s disease models. PPARα agonists have shown similar beneficial effects in rodent models of Parkinson’s disease and brain injury.
Investigators at Cornell have discovered that the administration of bezafibrate or combinations of PPAR agonists improved behavioral impairments, neuronal loss, and prolonged survival in mouse models of Huntington’s disease. Importantly, administration of bezafibrate increased numbers of mitochondria in both brain and muscle. The investigators also found that bezafibrate treatment improved the behavioral impairments and tau aggregation in mouse models of tauopathy.
We seek a licensee to develop these repurposed compounds, or an entrepreneur willing to start a company to develop them.
Transgenic R6/2 Huntington’s mice fed bezafibrate show an improved phenotype and extended lifespan. Kaplan–Meier survival plot of R6/2 mice on the bezafibrate diet in comparison to R6/2 mice on a standard diet. No wild-type control mice fed bezafibrate or a standard diet died in the observed time frame (figure not shown). N = 10 in each group.
- *Licensing
- Vibhu Sachdev(212) 746-6187sachdev@cornell.edu
- Country/Region
- USA
