New drug shows promise for motor neurone disease treatment

A pioneering study published in the International Journal of Molecular Sciences by researchers at Durham University has revealed promising results for a new treatment targeting motor neurone disease (MND).

The novel drug compound, Ellorarxine, developed by Nevrargenics, has demonstrated significant potential in improving neuronal health and nervous system regeneration in MND sufferers.

The study has shown that Ellorarxine exhibits antioxidant and anti-inflammatory effects at remarkably low concentrations.

One of the most exciting aspects of this new drug is its ability to address what researchers call the "3Ns" - neuroprotection, neuroplasticity, and neurorepair.

This comprehensive approach is rarely seen in potential MND treatments and could represent a significant advancement in the field.

Researchers observed that Ellorarxine increases MAP2 expression, a protein crucial for neuron growth and function. The drug also induces neurite outgrowth and improves mitochondrial viability, both of which are essential for maintaining healthy neurons.

These promising results have led to the compound receiving approval from the Medicines and Healthcare products Regulatory Agency (MHRA) to proceed to human trials, marking a significant milestone in its development.

Motor neurone disease, which affects over 5,000 people in the UK at any given time, is characterised by progressive muscle atrophy.

While current treatments only address symptoms, Ellorarxine shows promise in tackling the underlying causes of the disease.

This study is another strong indication of how promising our novel drug Ellorarxine really is.

It moves us closer to achieving our goal of bringing a drug to market and is testament to the power of conducting scientific research simultaneously across institutions to accelerate drug discovery."

Andy Whiting, emeritus Professor at Durham University and CEO at Nevrargenics

Professor Paul Chazot of Durham University, lead supervisor of the study, added: "I have been looking for a drug like Ellorarxine for over 30 years.

"It addresses the '3Ns' which provides a rational solution to combatting the complexity of neurodegenerative diseases, including motor neuron disease."

This research builds on a recent study published in Frontiers in Neuroscience by academics at Aberdeen University, which showed Ellorarxine's ability to cross the blood-brain barrier and concentrate in the spinal cord.

This property is crucial for any effective MND treatment and further underscores the potential of Ellorarxine.

Source:
Journal reference:

"Evaluation of a Synthetic Retinoid, Ellorarxine, in the NSC-34 Cell Model of Motor Neuron Disease", (2024), O. Escudier, Y. Zhang, A. Whiting and P. Chazot, International Journal of Molecular Sciences. https://www.mdpi.com/1422-0067/25/18/9764

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