Exploration of Relationships and Formulations of Molecular Tweezers

Study Rationale:                   
A key mechanism leading to death of neurons in the brain is formation of toxic clumps of the protein alpha-synuclein. Therefore blocking the formation of these toxic clumps is a promising therapeutic strategy for prevention and treatment of Parkinson’s. We are developing novel chemicals called “molecular tweezers” that have been found to prevent the formation of alpha-synuclein clumps in pre-clinical models.

Hypothesis:
Though are current lead compound, called CLR01, showed a therapeutic effect in models, its pharmacological properties need to be optimized before initiating clinical trials.

Study Design:
We will test several chemical strategies to improve and optimize the pharmacological properties of CLR01, including its penetration through the blood-brain barrier and possibly its conversion from an injection to a pill.

Impact on Diagnosis/Treatment of Parkinson’s Disease:             
Successful completion of our project will bring us an important step closer to the initiation of clinical trials and closer to development of this therapy to slow or stop Parkinson’s disease.

Next Steps for Development:
If the project is successful, we hope be in a position to begin the experiments required by the FDA for approval of clinical trials and to obtain the investment needed for conducting these experiments.