Anavex Life Sciences Corp (AVXL)

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Blarcamesine to Treat ALS

Amyotrophic Lateral Sclerosis (ALS, "Lou Gehrig's disease") is a severe CNS disease, with all sorts of progressing, lethal symptoms. Most or all involve hyperexcitability of nerves. And a primary agent of those over-excited nerves is glutamate. In animal models of ALS, when glutamate can be suppressed, ALS symptoms are reduced. Clearly, profound suppression of glutamate in nerves would be a major therapeutic step for ALS.

For detailed information on the subject, look at any of the links that appear after entering "Glutamate and ALS" in an online search engine. Here's a bunch:
https://duckduckgo.com/?q=Glutamate+and+ALS&ia=web

As those who scrutinize the clinical results of blarcamesine in humans know, from the six girls in the early Rett syndrome study of blarcamesine for safety and tolerability, two significant outcomes, in a very short period, occurred: a) glutamate levels significantly declined, and b) gamma-aminobutyric acid (GABA) levels increased.

For ALS, this would be a treatment double-whammy. First, sufficiently suppressing glutamate levels would turn off the core, rood-cause factor for ALS nerve hyperexcitability. This would be helpfully facilitated by elevation of GABA, which turns down pathogenic hyperexcitability in nerves.

There is no reason whatsoever that blarcamesine shouldn't be tried in humans with ALS, in a new clinical trial. First, as shown in all the other trials with real humans, blarcamesine produces no untoward, disqualifying adverse events ("side effects"). The drug, uncommon for those acting in the central nervous system (CNS), does not disrupt or adversely affect either the CNS itself, nor other body systems, organs, or tissues. Profoundly safe.

So, let's get some blarcamesine into ALS patients, to see if it can, as it does in Rett, suppress glutamate levels.

There is every reason to believe this would happen. Of even greater significance might be the very early administration of blarcamesine to ALS patients, when the disease is first detected; before it progresses to the various nerve and organ debilities that prove lethal. Again, prophylaxis, therapeutic "prevention" is likely to be profound.