Mitochondria-Targeted Therapeutics

With ongoing support from the Alzheimer Drug Discovery Foundation, we're conducting extensive drug discovery research to develop a preclinical drug candidate. Our project on mitochondria-targeted therapeutics discovery is directed toward the prevention and treatment of neurodegenerative diseases, particularly Alzheimer's disease.

Our Discovery Funnel includes multiple primary and secondary assays. These were specifically designed to allow a selection of the best mitochondria-targeted small molecules based on our understanding of the molecular mechanism of neuroprotection and the molecular target.

The Discovery Funnel is essential to develop safe and effective treatment because it allows research to develop molecules with high specificity and selectivity to the target. Other assays in the Discovery Funnel confirm whether new molecules penetrate the blood-brain barrier and could be safe for use in people.

Final validation of new compounds in a transgenic mouse model of Alzheimer's disease provides data on safety and effectiveness in cognitive protection. Dr. Trushina and her colleagues have demonstrated that partial inhibition of mitochondrial complex I activity using small molecules induced positive metabolic adaptation and a resistance to oxidative stress in primary mouse and human neuronal cells and in multiple mouse models of Alzheimer's disease.

Successful accomplishment of our mitochondria-targeted therapeutics project could provide a disease-modifying strategy for currently incurable conditions such as Alzheimer's disease.