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More and More. Another Test for Early Alzheimer's
Well, here's another team working on, among other things, new tests to detect Alzheimer's prodromally, before any symptoms appear, or before symptoms are ominous.
https://www.eurekalert.org/news-releases/984175
When blarcamesine gets approved, blarcamesine will be used to prevent the onset of Alzheimer's. A preventative, prophylactic therapy, not merely a treatment. Nothing better.
I Know the Right Research Team for This
HSP is rare, thankfully. Still, blarcamesine is needed.
A Second Blarcamesine MOA for HSP
Some time ago I posted my evidence-based conjecture that blarcamesine very likely will be a successful therapy for at least some forms, probably most, of hereditary spastic paraplegia (HSP), a rare disease where motor neurons, controlling body muscles, most prominently those controlling gait, in the legs, remain hyper-exited; constantly firing.
I have a genomically-confirmed case of HSP, with one of less severe genotypes. Severe cases become evident in childhood and muscles across the entire body are lethally spastic. Several dozen genotypes have now been identified, and mine is one of the less severe. Mine was primarily a middle-age on-set type. My legs stiffened in my 50s. Today, to walk I need the assistance of a walker; can’t go up or down staircases. But I can drive with complete safety and function. Wonderfully, there is no pain; just a confounding gait-inhibiting spasticity. (Well, it also caused a neurogenic bladder, so I surgically had to have created a suprapubic cystostomy, where I now have to contend with a suprapubic catheter.)
In severe cases (which it appears I will never attain), a fluid-filled sac containing baclofen is surgically placed in the spinal column, where the drug slowly perfuses into the long motor neurons extending to the adductor and other muscles of the leg, where the drug inhibits the continual firing of those nerves. Periodically, the sac is injected with make-up baclofen. A less than satisfactory treatment for severe cases of HSP. Thankfully, I have no prospect of needing the baclofen pump.
One understood mechanism of some or most forms of HSP is a deficiency of gamma-amino buytric acid, GABA, within neurons. GABA is an inhibiting neurotransmitter; keeping neurons from over-firing when exited by a nerve impulse. Nerves lacking sufficient GABA fire frequently or constantly; hence the spasticity of many or most forms of HSP.
Of course, the clinical trial of blarcamesine with girls with Rett syndrome revealed markedly increased, even restorative concentrations of GABA in their neurons. GABA-deficient Rett syndrome neurons account for the poor motor, muscle control of these afflicted girls. Blarcamesine’s ability to restore functional concentrations of GABA for them accounts, at least in one manner, for how the drug will be a useful Rett therapy.
From that clinical evidence, in real humans, I projected that blarcamesine would likewise be a successful treatment for GABA-deficient cases of HSP; for me, even. Eager to give it a try.
But, not all cases of HSP are solely GABA-deficiency diseases. It has just been discovered that dysfunctional calcium transport at the endoplasmic reticulum (ER) is also a cause of some or many forms of HSP. Very likely, both pathological mechanisms are involved.
The new research is here:
https://www.eurekalert.org/news-releases/983999
It’s a long and detailed report. “The results obtained indicate a new mechanism whereby the structure of the endoplasmic reticulum controls the synaptic function, and suggest a possible pathway by which it could cause hereditary spastic paraplegia.” In the simplest terms, restoration of calcium transport related to the endoplasmic reticulum should be profoundly therapeutic. Of course, blarcamesine activates the sigma-1 receptor protein, which then profoundly facilitates normalized calcium transport to and within the endoplasmic reticulum.
Now the form of HSP described in this paper is not one of the more common GABA deficiency forms. But, it now appears that two mechanisms of action (MOAs) of blarcamesine, both GABA synthesis and calcium transport at the ER, will be therapeutic for the disease.
HSP will be another human disease for which blarcamesine can yield safe, effective therapy.
Both monoclonal antibodies and Anavex molecules?
Well, the fellow seems competent in being able to assess and understand the several new therapeutic approaches to Parkinson's and Alzheimer's. He mentioned monoclonal antibodies, of course, but didn't specifically mention any agents working at the sigma-1 receptor protein (the Anavex mechanism).
However, I would expect him to mention not only Anavex's sigma-1 receptor activators but to explain in some detail how they work, as ligands of the sigma-1 protein, thereby facilitating or promoting a number of normalizing downstream processes and reaction chains in otherwise diseased neurons. Let's see if he recognizes and explains the Anavex MOAs (mechanisms of action). Those are the real story in new CNS therapies.
Let's see what he talks about.
Exactly. Anavex science threatens so many.
Private Anavex Research Can Surely Happen
Anavex NDA Will Be the Sigma-1 Biology Tutorial
Effective Sigma-1 Receptor Activation is the Story
Is Childhood Dementia the New Disease Target?
From Missling's personal presentation at this conference in Australia it seems very likely that the previously undisclosed new indication for blarcamesine may well be Childhood Dementia.
Information on the disease here:
https://www.childhooddementia.org/what-is-childhood-dementia
About one in 2800 babies born will suffer and die from the disease.
In 2021 3,664,292 babies were born in the US. At this rate a bit over 1300 would have died of the disease.
Contrary to the markets.
Right now, the AVXL share price is up 2.85% for the day, at $10.15. But the NASDAQ is down 0.43%.
This diverges markedly from recent share price trends. Most often, when the AVXL share price goes up a few pennies, the NASDAQ also appreciates. The reverse has been true. For many weeks, with few exceptions, the daily AVXL share price trends mirrored those of the NASDAQ.
But not today. Quite a divergence. Is something afoot? Today, what would be driving the AVXL share price so strongly against the declining NASDAQ?
It's just a bit after noon. Let's see if this continues for the rest of the day, and into tomorrow. Is something up with Anavex?
Good things will be discovered, first.
But why the recent increases?
Was Aduhelm "typical?"
What if Australia is first?
What the FDA should want.
You decide....
Anavex has several new drugs, beyond blarcamesine.
Watch. The FDA will get it "wrong."
Finally, an Anavex NDA is coming!
Well two of them, for sure.
The company will be hiring an intern to do biostatistics. Of course, the person will have to sign an NDA, a non-disclosure agreement. What he or she sees in the clinical results metrics will have to remain secret, non-disclosed --- until there's another NDA, a New Drug Application, which will use the worked-up statistics and go to the FDA to get blarcamesine approved.
All of which will be the final NDA --- No Doubts Actually
Qwerkiness (Well, Quwerkiness)
When the time is right.
Sab, I'm sure you are correct:
I must merely wait.
Earlier, I posted:
Presently, only for biostatisticians.
For anyone new coming to this message board to learn about Anavex Life Science Corp and its new drug candidates (blarcamesine, et al.), when reading through the majority of postings in the last week or more, you will be very frustrated.
Several years ago, the big question was whether or not the Anavex drug, Anavex 2-73 (now, “blarcamesine”) could possibly treat Alzheimer’s. Early clinical data showed both safety and efficacy, but since Alzheimer’s is so thoroughly recalcitrant (resistant to any treatment), the great question revolved around the drug’s mechanism of action (MOA); exactly, biochemically, how could it work its magic. How, possibly, could it work to yield positive therapeutic outcomes? On a molecular basis, what, if anything, was happening?
For several years the mechanism of action was the big question being discussed on this message board. Lots of “experts” laid out detailed reasons just why the molecule couldn’t possibly work any cellular fixes for neurons with Alzheimer’s. At the same time, other experts, with in vivo and in vitro evidence (in animals and cell cultures) showed that, indeed, by its unique activation of the sigma-1 receptor protein, all sorts of favorable, downstream reaction cascades happened, thereby restoring cellular homeostatic processes, etc.
If you wish, search back through all of the hundreds of postings in the last year or so, and you will no longer find any trying to prove that blarcamesine’s MOA is deficient. By now, the molecule’s remarkable abilities to fix various pathologies in neurons is uncontested. The drug works, safely and effectively. But now, to what extent?
Presently, the contentions focus on the statistics produced in human clinical trials of blarcamesine. It is contended that the biostatistics publicly released from the trials are not complete, so the actual safety and effectiveness of blarcamesine can’t yet be known.
Right now, to understand and participate in the message board’s current discussions one must have taken (and passed) and understood all that was in a college biostatistics course. Since very few retail stock investors have statistics competency, the current postings will provide little upon which the taking of an AVXL position can be decided.
By whatever means, sooner or later, the matter will be settled. Let’s see how.
Missling Machinations, for Ultimate Anavex Benefit
The MOA Not Questioned
The Faculty Here Are Failing. Market Truth Prevails.
Anavex had an online event before the market opened today, and then the share price went up 5.71% for the day. How could that have happened? All through the day, as every day previous, there were abundant postings on this board from real experts telling the frequent and many defects in Anavex science, clinical trials, and corporate leadership.
Only two explanations for this discrepancy; the Anavex is bad vs. Anavex has a future dichotomy.
The experts purveying their Anavex negatives, good people that they are, are simply not very good teachers. They are unable to clearly and simply convey the authentic deep truths of Anavex failures. They are sadly ineffective in conveying the truth. It’s there, but too detailed or complicated for general stock investor audiences to comprehend from our incompetent teachers.
Or, the investor audiences who come here to learn about Anavex, from what they’ve learned, now believe that Anavex actually has a future. Hence, shares were purchased and the AVXL price went up nicely today.
The naysaying faculty in this on-going “Introduction to Anavex Life Sciences Corp” course are going to have to do a better job. So far, they have presumed that their students are fellow faculty members and should understand, agree, and apply what they teach without question. Sorry. That’s not working right now, is it?
Of course the only truth that really counts is the truth of the current equity share price. Everything mentioned above is peripheral. What the inanimate “market” declares as the share price is the ultimate truth. Today’s real truth delighted most who visit this message board. They are glad that tuition rates are so low.
Just the science, only.
Oops. Unchecked exuberance.
Future Anavex Metrics
jmvho, those estimated, future Anavex metrics are very much in line with my calculations.
I, too, to be conservative began the number crunching presuming that Anavex would sell its drug(s) to only one million patients each year. My number ranges were very similar to yours.
But in truth, I believe both of us are being overly conservative. Frankly, when blarcamesine gets FDA approval to be used to treat (or prevent) Alzheimer’s, it will be used to treat far more than one million patients each year. Authoritative sources state that at least six million Americans suffer from the disease. If so, we can multiply our totals by 6x.
That’s just for Alzheimer’s, just in the US. What about Parkinson’s disease dementia? I can’t find a reliable number on how many in the US have this particular form of Parkinson’s disease, but apparently a million or more have conventional Parkinson’s in the US.
Of course, Alzheimer’s and Parkinson’s disease dementia are not confined to the US. Both diseases are global; but Anavex’s market will include the developed countries of the Western World, primarily those in North America and Europe. Revenues from blarcamesine sales outside of the US should at least equal domestic sales. So, multiply the revenue sums by 2x.
Understandably, people make good fun of the projected or anticipated eventual AVXL $1849 share price that appears on this message board. Right now, it’s good for some message board laughs and fun. But, in five years...?
Because of the MOAs.
Simple. Trade Secrets! Standard Biotech Practice.
No Buy Out — Just a Service Contract
“Disease Modifying” — What Does That Mean?
This phrase has been used in regard to Anavex drugs. But, what does it mean? I wanted to be sure, so I looked it up. On this particular webpage (http://jonathanharrismd.com/disease-modifying-therapy-what-is-it-and-how-do-we-know-it-works/) it said:
Will there be a squeeze?
But what will the shorts tell their wives?
If Anavex goes bust, I’m out a few thousand dollars. My wife will not be pleased, but will understand that I’ve not jeopardized the family’s finances. All lost dollars were from set-aside discretionary dollars intended from the start for “risky” investment ventures.
But what will the shorts be telling their wives?
Of course, if Anavex goes bust they can delight their wives with nice gifts and vacations, etc. They played the stock shorting game expertly. Well done. My congratulations and respect.
Or, what happens when, as just happened today, the AVXL share price begins a strong ascent, gaining new dollar values each week? Then, some sort of share price-driving info appears. The FDA likes an Anavex New Drug Application (NDA) and it looks like the company will be selling blarcamesine. With any sort of assured future revenue stream, today’s AVXL $10.87 share price will be distant history, never to be seen again. Shorts will be required to cover their positions; will have to buy at the market price real AVXL shares to “cover” the ones they “borrowed” at cheap prices.
There won’t be any congenial stock investment discussions between the adults in the families of AVXL shorters. Shorters, hope you can cover really soon, so things don’t go really bad for you.
What can I explain to my wife?
Early, Pre-symptomatic Diagnosis of Alzheimer’s
Here’s another one:
What counts more, real behaviors, or statistical datapoints?
Reasons for validity with 301 patients.