Pathways to protecting and repairing the central nervous system

Summary

Multiple sclerosis (MS) is a complex autoimmune and neurodegenerative disease with no clear cause and no known cure. People with MS indicate that new treatments to protect and repair the central nervous system (CNS) is their greatest unmet need.

Associate Professor Kaylene Young leads a multidisciplinary team of laboratory scientists, clinicians, biostatisticians, epidemiologists, geneticists, MS advocates and MS consumers, representing diverse experiences and vital perspectives to bridge the gap between MS research and practice. Their goal is to carry out laboratory research to identify signalling pathways that lead to the development of MS, learn how brain circuit function is impacted by MS, and design and translate treatments to protect and repair the brain. For this project:

1. The team will work with clinicians to progress their first potential myelin replacement therapy through a phase II clinical trial and evaluate its efficacy in people with MS;

2. Using laboratory models, advanced microscopy, behavioural analyses, and electrophysiology, they will investigate how myelin loss and replacement impact brain function;

3. They will study families with an unusually high incidence of MS, which may render clues as to MS associated genetics. The team will then use induced pluripotent stem cells to characterise the impact of rare, family-specific genetic variants on cell behaviour.

Progress to Date

Over the past year, Associate Professor Kaylene Young and her team have made significant progress in multiple research projects aimed at understanding and finding potential treatments for MS. In the phase I trial, transcranial magnetic stimulation (TMS) was found to be safe for individuals with MS, providing promising results. Moving forward, a phase II trial has been initiated with the involvement of six different sites, including Hobart, Newcastle, and Launceston. The Hobart site has already recruited its first participants, while recruitment is about to begin at the other sites.

In parallel, additional preclinical work has been carried out to investigate the cellular and molecular mechanisms underlying TMS-driven repair. These findings will be submitted for publication soon.

The team is also studying families with a high incidence of MS to identify genetic changes that play a role in the disease. The researchers are now exploring specific biological pathways using sequencing technology to identify genetically driven changes that contribute to MS susceptibility and progression. Furthermore, they are using cutting-edge technology to correct MS-associated genetic changes in stem cells generated from the blood of family members and have developed laboratory models carrying potentially detrimental genetic changes.

The progress made thus far has been impressive. The team has also welcomed new PhD students, and the early career researchers are experiencing excellent career progression. The involvement of MS consumers in research planning has been successful, and the team's achievements have been recognised, with one researcher being featured as a "trail-blazing woman in medical research" by NewsCorp Australia.

These discoveries provide hope for future interventions targeting myelin repair and neuroprotection. Additionally, ongoing work with stem cells and genetic changes offer valuable insights into the cellular changes associated with MS and potential pathways for therapeutic intervention. Associate Professor Young and her team’s dedication and progress demonstrate a promising path forward in understanding and addressing the complexities of MS.

Updated: 31 March 2023

Updated: 14 February, 2022

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