Using human genomics to identify drug targets for progressive MS

Associate Professor Justin Rubio

THE FLOREY INSTITUTE OF NEUROSCIENCE AND MENTAL HEALTH, VIC

January 2024

specialisation: Epidemiology

focus area: A cure via repair and regeneration

funding type: Fellowship

project type: Investigator Led Research

Summary

Multiple sclerosis (MS) is a common cause of neurological disability in young adults, affecting over 33,000 people in Australia and over 2.8 million people globally. There are treatments for relapsing forms of MS, but these are mostly ineffective in treating people living with progressive MS.

Associate Professor Justin Rubio and his team will study at genes from both single cells and human populations, to uncover any that may be involved in progressive MS and how they fit into the MS puzzle. Next, they will target these genes in human stem cells in the laboratory to understand their function in models of MS.

From this research, the team hope to discover potential drug targets for further investigation. The ultimate goal is to test newly developed drugs in clinical trials for progressive MS.

Progress

As a first step, Associate Professor Rubio and his team have focused on immune cells in the brain called microglia, that may be important in progressive MS.

They developed new laboratory methods to purify these cells from post-mortem MS brain tissue. The next step is to explore the genes of these cells and how they work in a laboratory model, to understand their potential role in MS.

Over the next 12 months, Associate Professor Rubio and his team will continue to investigate genetic changes in brain tissue including microglia to identify genes that may be involved in progressive MS.

Updated 31 March 2025

lead investigator

total funding

$650,000

start year

2024

duration

5 years

STATUS

Current project

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

3+ years

Read More

Newsletter subscription

  • This field is for validation purposes and should be left unchanged.

Using human genomics to identify drug targets for progressive MS