Transcript - Flintbox

The use of Pan-PPAR Agonists for Treatment of
Tauopathies and Huntington’s disease
Inventor:
Flint Beal, MD
Chairman of Neurology and Neuroscience/Neurologist-in-Chief
New York Presbyterian Hospital-Weill
Medical College of Cornell University
PPAR and neuroprotection
• Well established field – 92 hits on
pubmed; 13 reviews
• Great review of pan-PPAR agonism by
Beal, 2008
• PPAR agonists are anti-inflammatory,
antioxidant, protect mitochondria
“Modulation of several molecular and pathophysiological
pathways simultaneously may be a better approach for
neuroprotection. This could be achieved by using a
combination of several pharmacological agents or,
preferably, with only one having pleiotrophic effects. PPAR
agonists have the ability to induce gene expression and
modulate several molecular pathways at the level of
transcription. Therefore, PPAR agonists or drugs acting on
transcription factor receptors are possible therapeutic
targets for neurodegenerative diseases. Further, additional
experimental, preclinical and clinical studies are necessary
using PPAR agonists to determine neuroprotective effects
on disease progression as well as on disease onset.’
What’s new?
• Reduction to practice in P301S mice
(tauopathy)
• Reduction to practice in HD mouse
Bezafibrate reduced tau pathology in P301S mice.
Bezafibrate rescued behavioral abnormalities in P301S mice
Bezafibrate
restores the
PGC-1α
signaling
pathway in
R6/2 mice
Bezafibrate improves the behavioral phenotype and extends survival in R6/2 mice
• Developmental status
– POC established in 2 mouse models with bezafibrate
• Intellectual Property
– Provisional application filed
• Publications
– 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.
Contact:
Bruce Toman, CLP
Technology Commercialization and Liaison Officer
CCTEC
418 E 71st Street, Suite 61
New York, NY 10021
ph: 212-746-6187
[email protected]