Motor Neurone Disease Treatment Breakthrough Found

University of Queensland
Man's hand holding a drug vial labelled R8Y up to the camera.

(Photo credit: The University of Queensland )

Key points

  • UQ researchers have developed a drug that provides a unique tool to study a hard-to-target immune receptor.
  • The international collaboration uncovered the molecular make-up of the receptor, opening new possibilities for anti-inflammatory treatments for neurodegenerative diseases.
  • A drug treatment for MND could be available for testing in MND patients within 5 years.

University of Queensland researchers have developed a drug that activates a hard-to-target immune receptor - a breakthrough which could lead to new treatments for neurodegenerative conditions like Motor Neurone Disease (MND).

In an international collaboration with the Indian Institute of Technology, The University of Tokyo and Sungkyunkwan University, the researchers were able to uncover the molecular make-up of a receptor found on the surface of many immune cells called C5aR2.

Professor Trent Woodruff , from UQ's School of Biomedical Sciences , said the new drug provides a unique tool to study C5aR2 in detail, opening new possibilities for anti-inflammatory therapies.

"With this knowledge, it's possible we could have an anti-inflammatory drug treatment for testing in MND patients within 5 years, possibly turning the disease into a long-term chronic condition rather than an acute terminal illness,'' Professor Woodruff said.

"The drug we developed as part of this study, called R8Y, is a molecule that specifically binds to C5aR2,"

"This allowed us to see the structure and function of the receptor which led to the discovery that C5aR2 does not behave like most other receptors.

"It is unable to couple to G proteins - a rare and important distinction which explains why it's been so difficult to target and why its role in inflammation has been so elusive.

"Using the drug we created, our international partners created a model of the drug bound to the receptor, giving us vital information around how the drug binds to the receptor and how the receptor signals."

Using this knowledge, the UQ team is now working to develop even better anti-inflammatory drugs for hard-to-treat neurodegenerative diseases like MND, Parkinson's and Alzheimer's.

Associate Professor Richard Clark said the achievement was only possible through the combined expertise at UQ and its international partners.

"This global effort demonstrates the power of cross-disciplinary and cross-border collaboration in tackling complex biomedical challenges," Dr Clark said.

"With this new ability to selectively target the C5aR2 receptor, we can develop safer, more effective drugs with fewer side effects.''

Not-for-profit foundation FightMND provided funding towards the research.

"FightMND is proud to support this important research,'' Cure Research Manager Dr Michelle Kouspou said.

"We look forward to seeing this research advance and bringing us closer to better treatments for people living with the disease.''

The research was funded by FightMND and a National Health and Medical Research Council grant.

Read the research published in Molecular Cell.

Collaboration and acknowledgements

This project was conducted in collaboration with the Indian Institute of Technology, The University of Tokyo, Sungkyunkwan University, Pompeu Fabra University, Tohoku University and Kyoto University.

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