Vesalic targets ‘toxic exosome’ identified in ALS patients, suggesting disease biology may not be confined to the brain and CNS.
British biotech Vesalic has emerged from stealth claiming a potential breakthrough in how motor neuron diseases, and potentially other neurodegenerative diseases, can be understood and tackled. The London-based company says it has identified a “previously unknown” systemic metabolic dysfunction – found largely outside the brain and central nervous system – that appears to drive damaging processes in amyotrophic lateral sclerosis, the most common form of motor neuron disease.
According to Vesalic, the ALS field has spent decades pursuing targets in the brain and CNS with limited therapeutic success. If borne out, the company’s discovery suggests that at least part of ALS biology may be initiated and sustained by systemic processes rather than being confined to pathology within the brain and spinal cord.
“Years of focusing on brain and CNS-specific targets have been largely unfruitful in the search of both biomarkers and therapies for ALS and other neurodegenerative diseases,” said Vesalic’s Chief Scientific Officer, Professor Thomas Voit, also a Vice Dean at University College London. “It’s vital that we explore new scientifically driven hypotheses, including potential systemic pathogenic drivers. They could hold the key to unlocking desperately needed advancements for patients impacted by these diseases.”
Vesalic’s breakthrough is focused on exosomes, tiny particles that circulate in blood and carry molecular cargo between cells. The company’s researchers have identified what they describe as a “toxic exosome cargo” present in people with ALS. It appears this cargo originates from a broader metabolic dysfunction in the body, travels through the bloodstream to the central nervous system, and then binds to neurons and contribute to neuronal injury.
Rather than attempting to repair neurons after injury has occurred, Vesalic is developing an approach intended to neutralise the toxic cargo carried by exosomes before it reaches and harms motor neurons. The company is currently running in vivo studies aimed at establishing preclinical proof of concept, targeting clinical trials by 2027.
In parallel with its therapeutic work, Vesalic also working on a blood-based diagnostic test for ALS, after finding that the disease leaves a detectable fingerprint in lipids. The company says it has discovered a disease-specific alteration in the lipid composition of exosome membranes circulating in the blood. Based on this observation, Vesalic has developed a biomarker-based technology that it claims can detect cases of ALS with greater than 90% accuracy.
“Pushing boundaries to help us understand the causes and biological signatures of ALS is critical to delivering true progress against this devastating disease,” said Professor Kevin Talbot, Head of Clinical Neurosciences at the University of Oxford. “It’s incredibly exciting to see Vesalic advancing this therapy that could potentially address sporadic and monogenic ALS, alongside a simple, non-invasive biomarker test that could allow patients to be diagnosed much earlier.”
The company is also exploring the potential of exosome-lipid biomarkers in other neurodegenerative diseases, including Alzheimer’s and Parkinson’s, suggesting a broader hypothesis that its approach may be informative across conditions that have traditionally been framed as primarily CNS disorders.
“Our groundbreaking discoveries could fundamentally reshape the landscape in diagnosing and treating ALS, as well as other neurodegenerative diseases,” said Vesalic CEO Dr Valeria Ricotti. “We’ve made remarkable progress advancing our ALS therapeutic programme, our biomarker technology, and building our patent estate.”
