Monday, July 25, 2016 8:15:08 PM
An intelligent poster (above) asked me to tell the difference from Anavex 2-73 and all the previous Alzheimer’s drugs by other pharmaceuticals. One shouldn’t be investing in AVXL without knowing, at least superficially, those gigantic differences.
Presently, a lot of investment wags — who obviously couldn’t name off-hand three organelles (cellular components) — are ignorantly lumping 2-73 in with all of the several Alzheimer’s drug failures, along with the current acetylcholinesterase inhibitors (such as Aricept), which have minimal real usefulness. They improve cognition slightly for a short period of time, after which the disease continues on to to utter debility and death.
And that’s important to know. If, as it will, 2-73 exhibits even slightly better or longer cognition improvements, FDA will measure it against Aricept. No competition. Given 2-73's infrequent and minor adverse events — side effects — the FDA must approve it. It works better, and has fewer adverse events. Anavex has no real competition.
Why? Because all of the previous Alzheimer’s drugs have targeted different cellular functions, compared to the root-cause target of Anavex.
One big drug effort (don’t recall the name, offhand) attempted to prompt the immune system into clearing amyloid-Beta wastes. These protein waste clumps are microscopically diagnostic indicators of Alzheimer’s. And, clearly, they do disrupt proper and normal neuron function. It was thought that by clearing the a-Beta clumps, symptoms would resolve.
Except, no one was really able to prompt the immune system to only clear the a-Beta wastes. Other things in the brain were also affected, very severely. Immunotherapy approaches to treating Alzheimer’s have universally been a failure. I don’t believe any Big Pharma is current researching this any more.
A second approach, still targeting a-Beta wastes, is to find some molecule that a) crosses the blood/brain barrier (a difficult task), and then b) selectively enters affected neurons and somehow prompts the molecular clearing of a-Beta proteins. My understanding is that there might be several labs working in this arena. But the hurdles are high. Adverse effects will be obviating. I will stay clear of any such approach. Chances of therapeutic success extremely low, if not absent.
The current and only pharmaceutical treatment, such as Aricept, involves the administration of acetylcholinesterase inhibitors. These work outside the neuron, at the inter-neuron gaps, the synapses. The normal enzyme acetylcholinesterase breaks down an essential nerve signal chemical. By doing so, nerve impulses are facilitated across the synapses of Alzheimer’s neurons. But only for a few weeks or months. Acetylcholinesterase inhibitors soon lose their ability to keep things functioning. The disease then progresses degeneratively.
I know about this, as my father died of Alzheimer’s, and was a test patient in a clinical test for an acetylcholinesterase inhibitor drug. We had about three weeks of mild cognitive improvement, with no long-term good results.
Now, how is 2-73 different and so much better? On the face of it simple (but at the molecular level, rather complicated; I won’t attempt to present the deep molecular biology).
In virtually all neurodegenerative diseases, including Alzheimer’s, Parkinson’s, and others, neurons fail to function properly because reaction-controlling enzymes become mis-folded or physically distorted. Virtually all chemical reactions in cells are controlled by moderating enzymes. If any of those go wrong, disease ensues. In the case of Alzheimer’s normal nerve cell wastes don’t get cleared, and the diagnostic a-Beta waste protein clumps, along with Tau-tangles, accumulate. With all of that, nerves don’t function well.
Unlike all the others, Anavex targets the root cause of these debilities: misfolded or inadequate enzymes. And those are caused when the endoplasmic reticulum, responsible for protein folding, among other things, becomes physically or chemically detached from the cell’s power plant, the mitochondrion. Cells, with normal ER/mitochondrial connections function well. Disconnect the ER from the power-providing mitochondrion, and all sorts of cellular things will go awry.
In the simplest terms, 2-73 re-connects the endoplasmic reticula to the associated mitochondria. The cell then resumes normal function. Proper enzymes are made, wastes are cleared, and nerve impulses are transmitted unhindered.
And, remarkably, all of that happens at very low concentrations (doses between 30 and 50 mg), after the molecule rather magically jumps the blood/brain barrier and easily enters the neuron.
That, alone, is wonderful. But, most importantly, 2-73 appears to restrict its activities to the ER/mitochondrion connection, yielding few adverse events, none of which will obviate clinical use of the drug.
Almost too good to be true. But the biology is now known and demonstrated. It’s all up to the FDA to approve the drug. It has no competition. Nothing comes close.
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- Form 8-K - Current report • Edgar (US Regulatory) • 07/30/2024 09:20:53 PM
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- Form S-3 - Registration statement under Securities Act of 1933 • Edgar (US Regulatory) • 07/29/2024 09:21:49 PM
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- Contact The Gross Law Firm by May 13, 2024 Deadline to Join Class Action Against Anavex Life Sciences Corporation(AVXL) • PR Newswire (US) • 04/19/2024 09:45:00 AM
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