NeuroSense Therapeutics Ltd. (Nasdaq: NRSN), a company dedicated to developing treatments for severe neurodegenerative diseases, has announced promising preliminary results from a new biomarker study designed to evaluate the potential of their combination platform therapy for the treatment of Parkinson’s disease (PD).
By utilizing neuronal derived exosomes, the study compared blood samples from 30 healthy individuals to 30 people living with PD and identified modulations in PD-associated biomarkers, including AG02. These results hold great promise for the potential of NeuroSense’s combination therapy to provide new hope for those suffering from PD.
AGO2, the catalytic subunit of the protein complex that drives RNA-induced silencing, is a crucial player in the regulation of alpha-synuclein (αSyn) accumulation in the substantia nigra pars compacta – the region of the brain responsible for motor control.
Dysfunction of AGO2 can lead to a disruption in αSyn levels, potentially resulting in Parkinson’s Disease (PD). An understanding of the role of AGO2 in this process is essential to develop potential treatments for PD.
In a groundbreaking PD biomarker study, NeuroSense discovered an unexpected pattern: levels of AGO2 were significantly decreased in newly diagnosed PD patients (n=15) when compared to healthy controls (p=0.002). Interestingly, no significant changes were observed in AGO2 levels of more advanced stage PD patients, suggesting this could be a marker of disease onset.
In a Phase 2a clinical trial involving people living with ALS, NeuroSense’s platform combination therapy was found to have a positive effect, as evidenced by a statistically significant increase of AGO2 (p= 0.039) and a trend of increased levels of LC3 (p= 0.054). This promising therapy could be a potential breakthrough in treating ALS.
Recent studies conducted by NeuroSense have revealed that the levels of the essential autophagy protein LC3 are significantly lower in newly diagnosed Parkinson’s disease (PD) patients (p= 0.034). Autophagy is a cellular recycling process that is involved in the degradation of aggregated proteins and damaged organelles, and has been linked to a variety of neurodegenerative disorders.
This finding suggests an impairment in the recycling of damaged cell components in PD, and further studies are needed to explore its potential as a PD biomarker. Other PD-specific biomarkers were measured, but no trends were observed, so further research is needed.
The results of NeuroSense’s study strengthen the scientific rationale to develop its platform technology for Parkinson’s Disease (PD). The mechanism of action suggests targeting disease-specific pathways, particularly AGO2 dysregulation.
To further understand the involvement of biomarkers in the progression and pathology of PD, NeuroSense will continue to evaluate and explore these findings in future studies.
Prof. Roy Alcalay M.D., Chief of the Movement Disorders Center at Tel-Aviv Sourasky Medical Center, is thrilled to be part of this ground-breaking biomarker study for Parkinson’s Disease. He looks forward to furthering the research with this collaboration.
NeuroSense’s platform technology has demonstrated promising results in targeting early stages of Parkinson’s disease, a feat that could have a major impact in the fight against this debilitating condition.
Excited by these results, NeuroSense’s CEO Alon Ben-Noon has announced that the company is actively seeking collaborators to co-develop their PD asset in the hopes of providing earlier detection and improved treatments. It’s clear that NeuroSense is committed to making a real difference in the lives of those affected by PD.
About Parkinson’s Disease
PD can be a difficult condition to live with, affecting nearly 1 million people in the U.S. and an estimated 10 million people worldwide. Symptoms can include uncontrollable movements, balance and coordination difficulties, fatigue, depression, and memory difficulties, caused by the death of dopaminergic neurons in the substantia nigra. The PD treatment market is forecasted to reach $10.4 billion by 2031, representing a 6.2% CAGR and highlighting the impact that this condition has on the lives of millions.
AGO2 is a gene essential for miRNA function, and its disruption can have drastic effects on alpha-synuclein (αSyn) biogenesis. In cell and tissue cultures, depletion of AGO2 causes an accumulation of αSyn, leading to an increase in its mRNA and protein expression. This dysregulation of αSyn has been linked to Parkinson’s disease (PD) in pre-clinical studies, making AGO2 an important target for research in PD.
Autophagy plays an essential role in maintaining the health of our neurons and preventing the onset of neurodegenerative diseases like Alzheimer’s, Parkinson’s, Huntington’s and ALS. Microtubule-associated protein 1A/1B-light chain 3 (LC3) is a key player in this process, serving as an adaptor protein for selective autophagy and a marker for autophagosome biogenesis and maturation. Therefore, LC3 has become a widely used indicator for understanding the dynamics of autophagy and its implications for neurodegenerative diseases.
NeuroSense’s biomarker study, utilizing neuronal-derived exosomes (NDEs) extracted from plasma, showcases the potential of NeuroDex’s ExoSORT™ technology. By identifying NDEs and their cargo in bodily fluids, NeuroSense is on the cusp of discovering new biomarkers for prognosis and therapy. This is incredibly important as these vesicles can pass the blood-brain barrier (BBB) and offer a glimpse into the current state of neurons in the brain.
NeuroSense Therapeutics, Ltd. is a clinical-stage biotechnology company on a mission to discover and develop treatments for those suffering from debilitating neurodegenerative diseases. From ALS to Alzheimer’s and Parkinson’s, NeuroSense is determined to tackle some of the biggest challenges in modern medicine, where therapeutic options are still limited.
With a focus on the complexity of these diseases, they are committed to developing combined therapies that target multiple pathways to support the fight against neurodegenerative disorders.