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

focus area: Better treatments

funding type: Project

project type: Investigator Led Research

Summary

In 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 demyelination that occurs in MS causes loss of brain and spinal cord function and eventually brain and spinal cord degeneration.

Myelin is comprised mostly of lipids (fats), a large and diverse set of molecules that form biological membranes. Using mass spectrometry technology, Associate Professor Anthony Don and his team can measure hundreds of lipid molecules in the blood. They have shown that levels of lipids can not only differentiate people with MS from those without MS, but 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

Associate Professor Don and his team analysed the levels of about 150 different lipid molecules in 550 blood samples from a large international sample set. Their preliminary findings are:

  1. There is a distinctive lipid “signature” in the blood of people with MS, regardless of MS type.
  2. Certain lipid molecules in the blood correspond to neurofilament light chain, an established marker of damage to the brain and spinal cord.

This is important, as this blood lipid analysis method could be developed into a simple clinical test for myelin damage and other aspects of damage in the brain and spinal cord in people with MS. A simple test would help with monitoring disease progression and establishing whether a particular treatment is working for a particular person. Associate Professor Don is currently preparing a paper for publication in a peer-reviewed scientific journal.

The project is entering its second year and data analysis is continuing. Over the next year, Associate Professor Don and his team will search for lipids that correspond with lesion development, loss of brain volume and disease severity. They will also study whether lipid levels can predict disease stability or worsening in MS.

Last updated: 31 March 2025

lead investigator

co-investigator

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