Masitinib, a promising tyrosine kinase inhibitor, has shown potential to revolutionize neurodegenerative disease (NDD) treatment by addressing one of the most elusive challenges in neuroscience: halting neuronal damage. This new research, led by AB Science SA and published on bioRxiv, delves into how masitinib might mitigate neurodegeneration. Researchers targeted a model of experimental autoimmune encephalitis (EAE) to assess the drug’s effectiveness in reducing serum neurofilament light chain (NfL)—a key biomarker for neurodegenerative disorders.
The research tackled a significant problem: monitoring and reducing neuronal damage. Elevated levels of NfL in blood indicate axonal lesions and degeneration linked to conditions like multiple sclerosis, ALS, and Alzheimer’s. By focusing on reducing these levels, scientists aim to limit neurodegenerative diseases’ impact. In their study, researchers administered masitinib to EAE-induced mice, noting a remarkable reduction in NfL levels and pro-inflammatory cytokines over a 15-day period. This means masitinib could have neuroprotective effects, potentially modifying disease progression.
What makes this discovery significant is its broader implication. Patrick Vermersch, MD, a co-author of the paper, emphasized that lowering NfL indicates reduced neuronal damage and inflammation, which aligns with masitinib’s mechanism of action. This positions masitinib as a potential game-changer in neurodegenerative diseases with chronic neuroinflammation. Meanwhile, Alain Moussy, co-founder and CEO of AB Science, pointed out the significance of NfL as a biomarker recognized by regulatory agencies. With this recognition, masitinib’s influence on NfL levels may expedite approvals and accelerate the drug’s path to market.
Additionally, the treatment led to less grip strength deterioration in EAE mice compared to the control group, indicating potential functional improvements. This aligns with masitinib’s impact on pro-inflammatory cytokines and suggests it could slow the progression of neurodegeneration. By reducing inflammation, masitinib may offer hope to those battling progressive multiple sclerosis, ALS, and other related diseases.
In summary, masitinib’s ability to decrease NfL in an animal model provides a strong basis for further clinical investigation. This research brings hope that masitinib could become a novel treatment option for many neurodegenerative diseases, offering improved quality of life and a better understanding of disease progression. Its disease-modifying activity, combined with regulatory encouragement for biomarkers, positions masitinib as a promising contender in the fight against these devastating diseases.
