Anavex Life Sciences Corp (AVXL)

[falconer66a]

Temporary, short-period benefits; or permanent ones?

...the effect of simufilam is temporary, and then patients continue to decline -- similar to (but presumably safer and a bit more efficacious than) Aricept.

This is the understood effect, therapy, of Simufilam; understandably.

The goal is to have this new drug slow, even better than Aricept, the progression of Alzheimer’s symptoms. Neither of these drugs is intended to, nor can actually stop symptomatic progression; merely slow it. An Alzheimer’s diagnosis is lethal. There is no drug, presently, that can stop the disease’s lethal progression; merely slow for a period.

Not the case, however, with Anavex’s blarcamesine. It works very differently from Aricept or Simufilam. Those two drugs try to fix things “downstream” in the cascade of molecular anomalies that cause both symptoms and eventual death. For a time, they can slow these on-going pathologies, but the Alzheimer’s disease process continues, eventually resulting in utter debility and death.

Blarcamesine will either stop or actually reverse the Alzheimer’s disease process. That’s because it works “upstream,” at the start of the pathology, not downstream after the pathological processes are strongly in operation. It does this by attaching to and activating the sigma-1 receptor protein, which modulates, propels a number of consequent, downstream cellular maintenance functions, all of which, when taken together, obviate a diversity of CNS diseases.

First, with sigma-1 receptor activation, the synthesis of proteins, particularly enzymes, is properly facilitated. The resulting enzymes are properly-folded proteins, which can then catalyze chemical reactions efficiently and completely. Mis-folded enzyme proteins are a recognized cause or factor in most CNS diseases, including Alzheimer’s.

Blarcamesine-activated sigma-1 receptors also support, promote efficient and effective autophagy in the neuron, where worn-out, discarded, rejected, or mal-functioning proteins and other cell parts are efficiently digested and removed. Autophagy is the cell’s waste disposal function. With diminished autophagy (as in aging), toxic and complicating wastes accumulate; the cell functions ever more poorly.

Then, “homeostasis,” where activation of the sigma-1 receptor protein allows the cell to properly and efficiently detect and then correct chemical malfunctions. “Homeo-” means “same,” “-stasis” means status, or condition. Maintenance of proper chemical and biological conditions in the cell are required. Blarcamesine does this.

Then, blarcamesine also propitiously modulates gene expression, effecting the opening of chromatin surrounding genes, allowing their DNA to be fully, properly expressed.

Blarcamesine does even other good things. But these are the functions that will allow it to be both an effective treatment for Alzheimer’s, stopping or reversing that disease’s progression (not merely delaying it), and also an Alzheimer’s prophylactic, a preventative drug.

Neither Aricept or Simufilam will be able to compete. Once blarcamesine is approved for Alzheimer’s therapy, both physicians and patients will preferably opt for it. The other two drugs will be relative failures.