Study Rationale: Overwhelming evidence indicates that mitochondrial dysfunction contributes to the development of Parkinson’s disease (PD). We have shown that activating the enzyme AMPK prevents neuron damage caused by mitochondrial dysfunction in a pre-clinical model of PD. Our goal is to improve the drug-like properties of a novel AMPK activating drug so it can progress to PD patient studies.
Hypothesis: We aim to improve the drug-like properties of a novel AMPK activating drug so it can progress to human studies.
Study Design: In this study, we will work with medicinal chemists to improve the drug-like properties, such as brain penetration, stability and potency, of our novel AMPK activating drug.
Impact on Diagnosis/Treatment of Parkinson’s disease: AMPK activators have the potential to reduce neurodegeneration in Parkinson’s disease by reducing mitochondrial dysfunction and death of neurons. Since all patients with PD have underlying mitochondrial dysfunction, all patients, regardless of primary etiology, are expected to be eligible and benefit from this treatment.
Next Steps for Development: Once we have improved the drug-like properties of our AMPK activating drug, the next step for development will be to test the safety and efficacy in a pre-clinical model of PD before progressing to IND-enabling studies in preparation for human studies.