Neurons derived from stem cells from people with Parkinson’s disease offer an opportunity to perform drug and target discovery. We completed a screen of 4,000 drug compounds in such neurons and identified multiple molecules against a target involved in lipid biology and mitochondrial function. Our ultimate aim is to achieve neuroprotection by correcting perturbations in cell physiology, including alterations in lipid biology and mitochondrial function, in the early stages of Parkinson’s.
We aim to confirm our candidate drugs rescue disease phenotypes in neurons grown in a dish, and demonstrate that the compounds can reach their target in Parkinson’s models to validate new brain-penetrant drugs as potential treatments.
We will first perform genetic experiments in stem cell-derived neurons to confirm the data from our screen. Next, we will test the most potent drugs to rescue a range of cellular deficits. Finally, we will show that some of our lead compounds are able to cross the blood-brain barrier and hit their target in a Parkinson’s model.
Impact on Diagnosis/Treatment of Parkinson’s Disease:
Our aim is to develop new small molecule drug candidates that can tested in human studies.
Next Steps for Development:
The next step in our work will be test if our new drugs can function in Parkinson’s models.