A New Hope for Neurodegenerative Diseases

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Next-generation therapies to clear TDP-43 from the brain.

The Challenge

Neurodegenerative diseases such as motor neurone disease, frontotemporal dementia, Alzheimer’s, Parkinson’s, and Huntington’s have a common issue: the presence of a ‘culprit’ protein called TDP-43 in the brain. This protein is located within brain cells, making it difficult for medications to reach and effectively target.

Project Overview 

The Florey Institute is pioneering the development of immunotherapies aimed to remove the TDP-43 protein in the hope of treating various neurodegenerative diseases. If successful, this will be the first time creating a therapy that is capable of delivering antibody genes across the central nervous system barriers into patient neurons, where they can target and suppress the problematic protein.

Project Outcomes

  • Screen 1,000,000 nanobodies to find the most effective candidates for targeting TDP-43.
  • Test on human neurons to assess their impact.
  • Evaluate their effectiveness in preclinical models by examining neuroprotection, neuroinflammation, and TDP-43 pathology.

Measuring Impact

Hearts and Minds measures its impact across six core categories developed by the Association of Australian Medical Research Institutes. Key highlights include:

Advancing Knowledge
Research Capacity Building
  • Diverse Team and Collaboration: The team includes members at various career stages, such as a Doctor, Associate Professor, and Professor. They come from different areas of the Institute, including dementia, neurotherapeutics, and motor neurone disease research. This project is also part of the $100k challenge, encouraging collaboration between teams to gather initial data.
Health Impacts
  • Patient Outcomes: Aims to improve treatment outcomes for individuals with, or at risk of neurodegenerative diseases.
Economic Impacts
  • Grant Applications: The data from this project is crucial for securing larger grants from the Australian Government and other major funding sources.

This project will support larger research initiatives, representing a significant step towards developing more effective treatments for various neurodegenerative diseases. 

Funding support from Hearts and Minds Investments, as nominated by Core Fund Manager, Caledonia.

This content was last updated in July 2024, for further information and updates, visit The Florey.