Small Molecule Drug Development

This project focuses on evaluating chemicals in Alzheimer’s models, aiming to discover new small-molecule drugs and assess their effectiveness in protecting the nervous system, especially the brain, from damage or degeneration.

The approach involves enhancing the activity of the proteasome, a cellular structure responsible for breaking down harmful proteins, as a potential strategy to clear toxic protein accumulations associated with Alzheimer’s.

The project concentrates on a specific protein, USP14, and a compound called IU1 that inhibits its activity, showing promise in reducing the build-up of harmful proteins related to the disease.

The goal is to further investigate and develop drugs targeting USP14 for potential use in Alzheimer’s treatment. Advanced techniques, including artificial intelligence, will be employed for initial screening, followed by testing in cellular and other models.

Small molecules are organic compounds, simpler in structure than their larger counterparts, which often regulate biological processes. They can pass through cell membranes, including the blood-brain barrier, reaching specific targets.

The research team has developed a novel method to alleviate cell dysfunction and promote the clearance of Alzheimer’s-related protein beta-amyloid. Additionally, they’ve identified a new small molecule using this model as an efficient screening tool.

This project is focused on assessing these chemicals in Alzheimer’s models. The primary aim of the project is to discover new small-molecule drugs and assess how effective a treatment is in protecting the nervous system, particularly the brain, from damage or degeneration.

Dr. Prashant Bharadwaj received an Artificial Intelligence Molecular Screen (AIMS) award from Atomwise. Inc., a US-based drug development company. An ECU- Deputy Vice Chancellor Strategic Scholarship was also awarded for this study.

Alzheimer’s’ Research Australia contributes to the project by supporting Dr Bharadwaj’s salary and providing world-class facilities.

The impact

Developing effective small molecule drugs is a significant priority in Alzheimer’s research and could be a valuable addition to the fight against Alzheimer’s due to its targeted action and flexibility in targeting the disease’s various components.