Study Rationale: Neurodegenerative disorders such as Parkinson’s disease (PD) affect the brain. Some of the drugs designed to treat PD have unwanted side effects in the rest of the body. To minimize these adverse effects, we have developed a system for restricting drugs to the brain, where the neurodegeneration occurs.
Hypothesis: We will assess whether we can restrict the action of drugs to the brain to avoid side effects in the body.
Study Design: We have designed an “active drug” that travels everywhere in the body and a “blocking drug” that remains outside of the brain. This two-part system allows us to restrict the action of the active drug to the brain. We have already developed these drugs and tested them in isolated cells. In this study, we will test the system in preclinical mouse models of PD. Because the approach requires administration of two drugs, we will need to determine the optimal doses for each drug and the frequency with which the drugs should be given.
Impact on Diagnosis/Treatment of Parkinson’s disease: This approach could provide a much needed solution to the problem of how to treat PD with drugs that have desirable effects in the brain but unwanted effects in the body.
Next Steps for Development: If we can demonstrate our approach to restricting the action of drugs to the brain works in mouse models of PD we will test the approach in other preclinical models.