Characterization, Optimization and Validation of a Preformed Fibril Pre-clinical Model of Parkinson's Disease
Pre-Clinical Alpha-Synuclein Models, 2016
New pre-clinical models have been developed to study the hypothesis that damaging forms of alpha-synuclein (protein involved in Parkinson's) spread across brain regions from a single point of origin. The current study's objective is to validate this novel model to develop standard operating procedures so that industry and academic groups can reproduce it.
Preformed fibril alpha-synuclein injected into a single brain region will reproducibly spread to adjacent brain areas and produce pathology that can be used as end points in pre-clinical efficacy testing.
Alpha-synuclein preformed fibrils will be injected into a single specific motor area. Over the course of six months, pre-clinical models will be evaluated on motor performance. At three time points, we will examine the brains for spread of alpha-synuclein and related pathology.
Impact on Diagnosis/Treatment of Parkinson's Disease:
Production and validation of a model of alpha-synuclein spread will prove valuable to understanding inherent mechanisms and thereby development of treatments aimed at preventing spread. Furthermore, production of such a platform will allow for evaluation of treatment effects on multiple brain regions simultaneously to address non-motor regions involved in Parkinson's.
Next Steps for Development:
Successful validation and implementation of this model will encourage wide spread use and thereby provide a platform for multiple investigators to use the same platform from which to benchmark novel treatment strategies to bring forth into the clinic.
Chief Scientific Officer at Atuka, Inc.
Location: Toronto, Ontario, Canada