This Anti-Epileptic Drug Has Been Recommended For MND Clinical Trial

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.

From ALS News Today:

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