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Our Third Strategic Priority for MND Research in Focus: Improving Translation

15 August 2023

Since the launch of our Research Strategy, Catalysing a Cure, we have been sharing more details about each of our Strategic Priorities. We have already published articles about the first two - Validating New Targets and Accelerating New Treatments - and now we’re bringing you a deep dive into the final priority, Improving Translation.

‘Translation’ is a word you may have heard researchers use, but do you know what it really means? This article aims to bring you everything you need to know about translation and why improving it is so important to helping us tackle motor neuron disease (MND).

From bench to bedside

All research in MND, from the study of the most basic biology of motor neurons right through to the development of new drugs, begins in the lab (“the bench”). Our ultimate aim is to bring new treatments for the disease into the clinic (“the bedside”) and make them available to people living with MND. Real progress happens when we can successfully make the jump from bench to bedside - this process is called translation.

Unfortunately, many potential treatments for MND that looked promising in the lab have not been successfully translated into the clinic. Although we have much to learn from these "failures", each one costs money and time, and ultimately dashes the hopes of the MND community. To improve the likelihood that new treatments will make it to the clinic, we need to improve the process of translation, and that’s why we’re making this a Strategic Priority.

Our Enablers to Improve Translation

Through a collaborative prioritisation exercise involving over 100 people from the MND community, we identified several key areas that, with investment, should help to improve translation, and ultimately improve the success rate of clinical trials in people living with MND.

1. Earlier Detection and Diagnosis

In the lab, researchers use models of disease that manifest MND symptoms at a well-defined time point e.g. the SOD1-ALS mouse model develops first symptoms of MND within the first few weeks of life. Researchers watch the animals closely and administer the treatment as soon as symptoms appear, giving the animals the best chance of survival.

In people, an MND diagnosis is often completely unexpected, and many people with the disease wait up to 12 months between first symptoms and a confirmed diagnosis. By this time, the disease has progressed substantially and treatments are less likely to be effective. We are exploring ways to detect and diagnose MND sooner so that people can start treatment earlier; a better reflection of the way animal models are treated in the lab.

2. Better Biomarkers

If doctors suspect you have suffered a heart attack, they will test the level of cardiac troponin in your blood. This “biomarker” is a protein released by damaged heart tissue and is highly specific for the heart muscle, so a high level of cardiac troponin is unlikely to mean anything other than heart damage.

In MND, we currently rely on clinical severity scores, such as the ALS Revised Functional Rating Scale (ALSFRS-R) as the main way to determine the state of someone’s disease, but these are subjective and prone to inconsistencies. We are investing in research to discover more reliable biomarkers for MND, which will improve our ability to test if new drugs are effective, and will increase our confidence in clinical trial data.

3. Patient Stratification

A diverse range of genetic and environmental factors are at play in MND, meaning the disease presents differently in different people. By grouping, or “stratifying”, people living with MND by different factors, specific sub-groups can be offered treatments that are more likely to be effective - a more “personalised” approach.

For example, the PRELUDE trial is testing the drug, lithium carbonate, only in people with a specific mutation in the UNC13A gene because research suggests the drug is more likely to be effective in people with this mutation. Previous clinical trials of lithium carbonate, that tested people with MND as one population, failed. Identifying new stratification techniques will help potential treatment effects to be identified more easily, so clinical trials will be more likely to succeed.

We recognise that these are just three of many barriers to successful translation. That’s why we are keeping an eye on other innovative ideas to improve translation, so that we can invest in any area that will help to ensure promising treatments make it to the clinic and are made available to people living with MND sooner. 

To help us continue to support research in this area, and all our Strategic Priorities, please visit this page to make a donation or explore ways to fundraise.

Our Third Strategic Priority for MND Research in Focus: Improving Translation
Our Third Strategic Priority for MND Research in Focus: Improving Translation

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