Ellorarxine shown to reduce neuronal death in neurodegenerative disease models

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A peer reviewed study published in Frontiers in Neuroscience by academics at Aberdeen and Durham Universities has shown that Ellorarxine, a novel drug compound developed by biotech spinout Nevrargenics, has shown a robust neuroprotective effect in cultured cells and reaches its target in animals. 

The cell studies involved two approaches, excitotoxic and proteostasis disruption assays. The excitotoxic assay was designed to model the ability of Ellorarxine to limit the effects of excitotoxic damage caused by neurotransmitters like glutamate, damage that may occur in neurodegenerative disorders such as motor neuron disease. The proteostasis assay was designed to evaluate the ability of Ellorarxine to help the cell get rid of dysfunctional proteins, a necessary function that is believed to decline as one of the causes of neurodegenerative diseases. 

Lead investigator Dr Azita Kouchmeshky, supported by Professor Peter McCaffery and Professor Andy Whiting, conducted a study using these assays to test the potential neuroprotective action of Ellorarxine for ALS and other neurodegenerative diseases. As expected, when the neurones were exposed to glutamate, they started to die. But treatment with Ellorarxine significantly reduced this cell death, suggesting the drug can protect neurons when exposed to such stress. 

In the second assay study, the team disrupted proteostasis in a motor neuron cell line. Again, treatment of this disrupted cell line with Ellorarxine significantly reduced this cell death, suggesting neuroprotective qualities. 

The in vivo animal tests showed Ellorarxine crossing the blood-brain barrier and inducing the expression of neuroprotective genes in the brains of mice. These factors, and the demonstrated concentration in the spinal cord, were pivotal in the company securing approval from the Medicines and Healthcare products Regulatory Agency (MHRA) to run a human study in the coming 12 months. 

The study demonstrated that the drug compound developed by Nevrargenics, Ellorarxine, provides a robust neuroprotective effect and reduces neuronal death in cell models of neurodegenerative disease. The researchers believe this neuroprotection should eventually lead to improved neuroplasticity and recovery.

Andy Whiting, emeritus Professor and CEO at Nevrargenics said: “This study is hugely promising and indicates the potential our novel drug compounds have in providing neuroprotection in neurodegenerative disease models, including ALS. The evidence generated by this paper was absolutely essential for securing the green light from the MHRA, which will see us move toward human studies in the very near future.”

Professor Peter McCaffery, lead supervisor of this study said: “These are the first tests of these novel compounds to show they protect and promote survival of neurons under conditions similar to neurodegenerative disease.  It’s very promising that not only are they active in these tests but are also able to reach the brain and spinal cord where they need to have their effect.”