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Small-Molecule Inhibitor of Alpha-Synuclein Propagation in the Treatment of Parkinson's Disease

Study Rationale:
The notion of misfolded alpha-synuclein (alpha-syn) aggregates moving from neuron-to-neuron is a mechanism that is gaining widespread acceptance as a central mechanism in the development of Parkinson's disease. Therapies designed to stop this spreading point towards a novel therapeutic route for preventing the development of Parkinson's disease.

With the development of a small molecule that impedes the spreading of alpha-syn aggregates, we propose to identify the target(s) of this compound, with the goal of understanding how it blocks alpha-syn spreading. These targets can then be exploited in future medicinal chemistry efforts to develop additional lead compounds.

Study Design:
As our small molecule was identified through a phenotypic screen, the cellular target(s) of this compound are unknown. Thus, our goal for this study is to identify these target(s) through a combination of complimentary genetic and proteomic approaches. This will allow us to narrow down the list of putative target genes for testing and validation as drug targets of our small molecule. We are confident that results from these distinct but complementary approaches will help inform each other, thereby guiding the direction of the work and increasing our likelihood of success in identifying the target(s) of our small molecule.

Impact on Diagnosis/Treatment of Parkinson's disease:
This proposal will allow us to rationally identify target(s) of our small molecule that can then be exploited in future medicinal chemistry efforts for the development of additional lead compounds in the treatment of Parkinson's disease.

Next Steps for Development:
Our goal is to move our compound and future analogues into Phase 1 trials. Such a trial, in partnership with CDRD and its industry partners, can be conducted at the MNI through its clinical research unit (CRU), which has ample experience in conducting such trials.


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