ALS researchers take ‘significant step’ toward new treatment

LIVERPOOL, United Kingdom — From baseball legend Lou Gehrig to the ice bucket challenge, the world’s awareness about Amyotrophic lateral sclerosis (ALS) has remained high for decades. Despite constant research however, there are few options which help treat the condition and none that cure it. Now, an international team says a new drug may finally help delay the death of nerve connections in ALS patients.

Scientists from the Universities of Liverpool (United Kingdom) and Nagoya (Japan) find a Selenium-based drug-molecule called ebselen and other compounds can change the toxic properties of a protein linked to ALS. A mutation in this protein, superoxide dismutase (SOD1), can cause ALS — especially cases believed to be passed through family genes.

Why is ALS so deadly?

ALS, also known as Lou Gehrig’s disease and motor neuron disease, is a degenerative disease which attacks the brain and muscles. This incurable condition slowly breaks down the nerve links between the brain and body, eventually causing paralysis. Most patients also lose the ability to speak without assistance and die within two and five years of their diagnosis.

About 20 percent of the familial ALS cases show a connection to dominant mutations in the SOD1 gene. Until now, only two drugs show limited results in treating these mutant proteins. Riluzole was approved for use in 1995 and edaravone in 2017.

“Choices are very limited for a current ALS therapy, therefore, we are excited to take a significant step forward for developing a new class of drug candidate for ALS,” Professor Koji Yamanaka of Nagoya University says in a media release.

How does ebselen work?

The new drug compound focuses on stabilizing the SOD1 structure. The team says in vitro therapeutic trials reveal ebselen is significantly more potent than edaravone. Experiments on mice show the drug clearly delays the onset of ALS, including the creation of ligand-bound A4V SOD1. This mutant protein is known to cause the most severe disease outcomes.

“The fact that this new generation of organo-selenium compounds have better in vitro neuroprotective activity than edaravone holds a significance promise for the potential of this class of compounds as an alternative therapeutic agent for ALS treatment,” study leader Samar Hasnain explains.

Hasnain adds these compounds could possibly have other benefits, including defending against viral diseases like COVID-19.

The study appears in the journal EBioMedicine.

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