Another interesting finding in the ongoing hunt for MND treatments comes from University of Tokyo researchers, who have discovered that a safe, existing anti-epileptic drug called perampanel has successfully prevented the death of motor neurons in mice with an animal model of MND.
Their research shows that perampanel is worthy of a clinical trial for MND treatment.
Unfortunately many other potential MND treatments that have shown great promise in mice have so far failed to work in humans – but the advantage in this case is that the drug is already approved and considered very safe, so a clinical trial is a much easier and less expensive endeavour than most.
In this study, researchers based their research on evidence of faulty mechanisms within motor neurons of ALS patients, especially the mislocalization of the TDP-43 protein, observed in the vast majority of ALS patients and considered a pathological hallmark of the disease. Researchers also focused on an RNA editing failure observed in the motor neurons of ALS patients.
Researchers used an anti-epileptic drug, perampanel, to repair this faulty mechanism in mouse models and used the normalization of TDP-43 mislocalization as a marker for the effectiveness of the treatment.
Experimental results showed the drug was able to prevent the death of motor neurons and normalized the localization of TDP-43 with the cells. Moreover, long-term administration of the drug in mice also led to visible improvement of motor dysfunction in the treated mice, as compared to the control mice.
Read the original research paper: The AMPA receptor antagonist perampanel robustly rescues amyotrophic lateral sclerosis (ALS) pathology in sporadic ALS model mice