Funded Studies
This grant builds upon the research from a prior grant: Exploiting a Novel Mechanism for Gene Regulation to Develop Innovative, Targeted Therapies for Parkinson’s Disease
Study Rationale: Parkinson’s disease (PD) and Alzheimer’s disease (AD) are neurodegenerative disorders caused by an accumulation of protein aggregates that leads to cell death. Genetic studies suggest that proteins that contribute to the pathology in one of these disorders may also be involved in the other. The protein Parkin, for example, has been linked to familial cases of PD. But some studies indicate it may also be involved in AD. We have developed compounds that increase the activity of Parkin and improve cognition. Here, we will test whether these compounds can help relieve some symptoms of PD.
Hypothesis: We hypothesize that compounds that enhance the activity of Parkin may relieve some symptoms of PD and help individuals suffering from forgetfulness, trouble with concentration and dementia.
Study Design: We will introduce chemical modifications in these compounds to make them more stable.
Impact on Diagnosis/Treatment of Parkinson’s disease: Our drugs have the potential to ameliorate memory problems experienced by people with PD. Our drugs will also treat some motor symptoms.
Next Steps for Development: Our next steps will be the preclinical testing of our most advanced compounds (IND-enabling studies) to determine whether they are safe for use in a human phase 1 clinical study; we will also improve our other compounds to make them more stable and active.