Developing a blood test to predict and track therapeutic response in MS

Associate Professor Anthony Don

The University of Sydney, NSW

January 2024

Specialisation: Epidemiology, Genetics

focus area: Better treatments

funding type: Project

project type: Investigator Led Research

Summary

In multiple sclerosis (MS), the immune system attacks and depletes the myelin sheaths that insulate and protect nerve cells. Myelin acts like insulation on copper wires and is essential for neurological function. The loss of myelin (demyelination) that occurs in MS causes loss of brain and spinal cord function and eventually brain and spinal cord degeneration.

Myelin is made up mostly of lipids (fats), a large and diverse set of molecules that form biological membranes. Using mass spectrometry technology, Professor Anthony Don and his team can measure hundreds of lipid molecules in the blood. They have shown that levels of lipids can tell people with MS from those without MS and also provide a readily accessible and accurate read-out of disease activity in a cohort of people with MS.

This project will determine firstly if levels of specific lipids in the blood serve as indicators of ongoing demyelination and disease progression in people with MS. Secondly, the project will determine if specific lipids serve as useful indicators of therapeutic response in MS. This is crucial because it means doctors could use a simple blood test to check if a treatment is working. Currently, doctors rely on brain scans and tests of brain function, which are done less often and can only spot problems after they have already happened.

The proposed blood lipid test is also needed for clinical trials of therapies that aim to regenerate myelin and restore neurological function in people with MS and which can be implemented on mass spectrometers that are already used in diagnostic pathology labs.

Progress

Professor Don and his team have measured the amount of about 120 different lipids in 550 blood samples from people with and without MS. They have successfully identified a set of around 25 lipid molecules that differ substantially in people with MS (both relapsing and progressive) compared to people without MS. But although these blood lipid levels differ, they cannot be used to definitively tell the difference between people with and without MS. Instead, these differences in blood lipid levels indicate that whole-body metabolism is altered in people with MS and this is possibly so that the body can control its immune system.

The team has identified certain lipid molecules that are linked to markers for brain volume loss and the loss of white matter (nerve fibres in the brain). They have also identified lipid molecules that may be able to predict disease worsening.

Over the next 12 months, Professor Don and his team will run final analyses of their data and prepare a detailed research publication later this year. They will focus on which blood lipids predict disease progression and/or white matter loss in people with relapsing or progressive MS. They aim to develop a blood test for myelin degeneration or ongoing disease activity. Such a test would help with monitoring disease activities and responses to treatments, improving clinical care.

publications

Shi et al, The Serum Lipid Profile of Relapsing Multiple Sclerosis Differs Reproducibly From Healthy Controls. Journal of Neurochemistry, 2025, 169:e70285.

Last updated 31 March 2026

lead investigator

co-investigators

Associate Professor Laura Piccio
Dr Ramon Landin-Romero
Associate Professor Todd Hardy
Dr Ellis Patrick

total funding

$249,995

start year

2024

duration

3 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

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Developing a blood test to predict and track therapeutic response in MS