Study Rationale: Some people with Parkinson’s disease (PD) have a change, or mutation, in a gene called GBA1. This gene makes an important enzyme called GCase, which helps keep brain cells healthy. When GCase doesn’t function normally, it can lead to a type of PD called GBA1-PD. People with this type of PD often get symptoms at a younger age and their disease tends to get worse more quickly. Right now, there are no medicines for these people, so urgent help is needed.
Hypothesis: Medicines that help GCase function properly could keep the brain healthier and make life better for people with GBA1-PD.
Study Design: Congruence is using two ways to help treat GBA1-PD and improve the chances of success. The first way is by making a medicine that helps increase the activity of GCase. The second way is by creating a medicine that helps GCase get to the right place in the cell, the lysosome, and work better once it’s there.
Impact on Diagnosis/Treatment of Parkinson’s disease: There is no approved treatment that can slow down or stop the progress of Parkinson’s disease. We are working to discover and develop new medicines that help the GCase enzyme function normally in people with GBA1-PD and hope these medicines will slow down disease progression.
Next Steps for Development: If we are successful in discovering the right molecules, we will first do experiments in the lab to check whether they work and are safe and then get approval to start a study in people with GBA-PD disease to see how well our medicine helps to slow down or stop the disease.