In Vivo Efficacy of a TRPML1 Activating Compound in Alpha-synuclein PD Pre-clinical Models

Study Rationale: Many genes harboring mutations that increase the risk for developing Parkinson’s disease (PD) are associated with the cell’s recycling center, the lysosome. Compounds activating the lysosomal protein TRPML1 has been discovered to improve the function of the lysosome, and we therefore believe that it could be a potential new therapeutic drug for PD.

Hypothesis: The TRPML1 activator can curb the disease development in pre-clinical models of Parkinson’s disease.

Study Design: We will test our TRPML1 activator in two mouse models of PD to see if the drug improves the symptoms in pre-clinical models. The effect of the treatment will be assessed by improvement in PD hallmarks such as pathological synuclein aggregation dopamine neurons and degeneration of dopamine neurons as well as motor dysfunction.

Impact on Diagnosis/Treatment of Parkinson’s disease: If we see beneficial effects of TRPML1 activator in the PD pre-clinical models, this suggests that the compound could have therapeutic effect in PD patients and would support further development of the compound as a therapy for PD.

Next Steps for Development: If successful we will continue the development of the TRPML1 activator drug by testing the safety of it in two pre-clinical models. This is to make sure there is no toxicity associated with the drug before dosing it in humans.