This grant builds upon the research from a prior grant: Using Small Molecules that Modulate Mitochondrial Function for the Treatment of Parkinson's Disease
Study Rationale: Mitochondrial dysfunction is a crucial driver of the initiation and progression of Parkinson’s disease (PD). Mitochondria are structures that produce cellular energy, and in neurons from people with PD they show deficits in energy production as well as cell signaling. Several of these deficits appear very early in PD progression. Rather than focusing on later-stage symptom modification, targeting early mitochondrial deficits offers a better chance to develop a disease-modifying therapy for many types of people with PD.
Hypothesis: We hypothesize that improving several key mitochondrial functions simultaneously will lead to an improvement or reversal of important dysfunctions seen in cells and neurons from people with PD and lead to the development of a disease-modifying treatment.
Study Design: Our team at LucyTx will test molecules it designs and makes to assess whether these compounds improve mitochondrial function in cells and neurons from people with PD or in cells that replicate PD pathology. Based on the results of these experiments, we will design additional molecules and optimize them to be suitable for clinical trials. We will also look for biomarkers or changes caused by our compounds which could be used to monitor impact on our target and improvement for PD patients in a clinical trial.
Impact on Diagnosis/Treatment of Parkinson’s disease: Mitochondrial dysfunction is implicated in both familial (genetic) and sporadic (idiopathic) cases of PD. Our mechanism of action suggests the potential for our drug to affect multiple types of PD and to act as disease-modifying therapeutics for both early and later stages of disease by targeting a nexus point of PD pathology.
Next Steps for Development: If our project is successful, LucyTx will have a candidate drug with early toxicological data suitable for Phase 1 clinical entry and we will have identified biomarkers which could help select participants for our clinical trials.