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A Project to Identify a Compound that will stop, and ideally reverse, Parkinson’s disease
Executive Summary
A five phase plan to identify one or more chemical compounds that may be able to prevent, slow, or possibly even reverse the aggregation of alpha(a)-synuclein. A successful project could lead to the discovery of a ‘neuroprotective’ compound that actually stops the progression of PD.
Based on recent advances in several areas, scientists and researchers at the Parkinson’s Institute (PI) in Sunnyvale, California, led by Dr. J. William Langston, a world renowned expert on PD, have become convinced the strategy outlined in the plan could lead to the discovery of a compound (drug) that would stop the aggregation of a-synuclein, protecting the cells from the progressive degeneration associated with the over-expression of a-synuclein. If this drug could break up, or disaggregate, the a-synuclein clumps, it might actually re-vitalize the dopamine-producing neuronal cells, reversing the effects of the progressive cell death and, therefore, PD.
The project’s five phases might be represented as follows:
| Phase | Principal Investigator/Collaborator | Compounds Tested | Model |
| I | Langston/Tony Fink, UCSC | 20,000-30,000 | test tube (in vitro) |
| II | DiMonte/Guy Caldwell, Alabama | 100-300 | C. elegans (worm)* |
| III | DiMonte/Ron Mandel, Florida | 30-100 | transgenic* mouse |
| IV | Dino DiMonte, TPI | 10 | pre-clinical study, genetically altered* |
| V | Caroline Tanner, TPI |
1? | small scale, Phase II or III clinical trial |
* modified to over-express α-synuclein
(Phase II through V would be performed at the Parkinson’s Institute.) The original compounds tested would already be FDA accepted as non-toxic to humans. We estimate this will cost approximately $5-6M and take two years to clinical trials.
As of June 2005, the first phase has been funded by a peer reviewed grant from the Michael J. Fox Foundation for Parkinson's Disease Research. As of August, 2005, a private foundation and private donor have both contributed to this initiative, bringing the total available to over $500K, a significant start.