A new therapeutic approach for brain repair in multiple sclerosis

Ms Danica Nheu

Monash University, VIC

| A cure via repair and regeneration | Neurobiology | Scholarship | 2023 | Investigator Led Research |
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Summary

MS is one of the most common, chronic neurologic diseases of adults worldwide, affecting more than 2.8 million people worldwide with 10,000 new diagnoses made each year. MS tends to strike early in adulthood, with women three times more likely than men to be diagnosed. The total direct cost to the US community of MS is just over $28 billion annually. 

MS is caused by the body’s own immune system mistakenly attacking the brain, spinal cord or optic nerves. The primary target of this attack is myelin, the protective coating around the nerve fibres, which carry nerve impulses between nerve cells. These attacks cause active MS lesions, and the nerve cells themselves can also be damaged leading to life-long disability. 

The research team, headed by Dr Steven Petratos, has shown that a modified version of a specific protein is present within active MS lesions in a laboratory model of MS. This modified protein then interacts with another protein to cause nerve fibre damage.  

The aim of Ms Danica Nheu’s project is to propose a new method to block either the modification or the interaction between the two proteins, to halt disease progression and provide recovery from disability. 

Progress to Date

Using a laboratory model, Ms Nheu has been able to preserve the fibre (axon) of optic nerves by deleting a protein in optic nerves called Nogo Receptor 1. Laboratory models show that damaged areas of the spinal cord and the optic nerve can be recovered. 

By using a new haematopoietic (blood) stem cell delivery system of a therapeutic molecule NgR(310)-Fc, Ms Nheu was able to overcome the issues that promote nerve fibre damage. This system is designed to reach MS disease areas only and maximise repair of the brain where the myelin is damaged.  

Ms Nheu is now investigating this molecule as a treatment and its ability to repair MS-like brain stem cells as a proof-of-principle trial. It is anticipated that this investigation will translate use of the molecule and system towards a clinical treatment.   

Publications

Ellen O, Ye S, Nheu D, Dass M, Pagnin M, Ozturk E, Theotokis P, Grigoriadis N, Petratos S. The Heterogeneous Multiple Sclerosis Lesion: How Can We Assess and Modify a Degenerating Lesion? Int J Mol Sci. 2023 Jul5;24(13):11112.  doi: 10.3390/ijms241311112. PMID: 37446290; PMCID: PMC10342336.  

Updated 31 March 2024 

Updated: 22 February, 2023

Stages of the research process

Fundamental laboratory
Research

Laboratory research that investigates scientific theories behind the possible causes, disease progression, ways to diagnose and better treat MS.

Lab to clinic timeline: 10+ years
Translational
Research

Research that builds on fundamental scientific research to develop new therapies, medical procedures or diagnostics and advances it closer to the clinic.

Lab to clinic timeline: 5+ years
Clinical Studies
and Clinical Trials

Clinical research is the culmination of fundamental and translational research turning those research discoveries into treatments and interventions for people with MS.

Lab to clinic timeline: 1-5 years

Investigator

Supervisor

Total Funding

  • $105,000

Duration

  • 3 years – starting 2023

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A new therapeutic approach for brain repair in multiple sclerosis