During 2024, encouraging research was published by the Universities of Durham and Aberdeen that demonstrated that a new drug, Ellorarxine, was able to reduce the death of rodent neurons grown in a dish, including those containing an alteration found in some forms of MND. 

This is a welcome finding that further informs our understanding of the mechanism underlying MND and reveals a new target and drug for possible intervention and modulation. 

Ellorarxine is a new compound, which is being developed by the company Nevrargenics. The published research indicates that it works by activating the retinoic acid receptor (RAR) system, which plays a critical role in a number of important activities throughout the body, including regulating cell development and survival. 

While this is an encouraging development, the research is still at an early stage and before Ellorarxine, which is a newly created compound, can be tested in people with MND it needs to be shown to be safe. This is initially done via a series of tests in the laboratory, which examine the toxicity of the drug in different models to determine the safest concentration that might still be effective. Only then can it be tested in people. 

Once there is a body of evidence from laboratory studies to indicate that a drug might be effective in treating a condition at a dose that isn’t toxic, then permission can be granted for a Phase 1 clinical trial.  This is an important first step that helps to determine whether a new compound is safe in humans – this stage is usually, though not always, undertaken in a small number of healthy people. A phase 1 trial usually takes less than a year to complete and should be automatically listed on the UK Clinical Study Registry.

MND Scotland, the MND Association and My Name’5 Doddie Foundation recently produced a series of infographics to explain the pathway a new drug needs to navigate before it can become a treatment. This can be found here.