Replies to post #331444 on Anavex Life Sciences Corp (AVXL)

[Steady_T]

I thought the voucher was only for rare pediatric diseases. There is a voucher program for tropical diseases and I don't think Anavex would be after one of those.

[Steady_T]

The orphan disease program is a different program. That gets the applicant....

 Orphan drug designation qualifies sponsors for incentives including:  
  
 Tax credits for qualified clinical trials 
 Exemption from user fees  
 Potential seven years of market exclusivity after approval

[falconer66a]

A candidate family of blarcamesine rare diseases.

As I’ve contended before, there is a group of closely-related rare diseases for which blarcamesine is very likely to be profoundly therapeutic, even prophylactic.

As a biologist, I’ve studied these closely. I, personally, have one of the diseases.

The family of diseases are those included in HSP, hereditary spastic paraplegia (of which there are a wide diversity of genotypes and phenotypes), and primarily lateral sclerosis (PLS).

In all of these, neurons fail to function properly. Most often, neurons controlling muscles (motor neurons) become hyper-excited, firing often, strongly, or continuously. That causes the spasticity, the continuing contraction in various muscles.

Personally, I have a very mild, late-onset version of HSP—which, in fact, was not hereditary. Many cases of HSP are actually spontaneous, not genetically inherited; merely an incidental mutation. Neither of my parents had HSP, and neither of my children inherited it; thankfully. In my case, the motor neurons controlling the adductor muscles of my legs (and a few other, minor ones, especially in the bladder—now have a suprapubic catheter) are continuously taught. I can walk, with a walker; am able to drive a car very safely and effectively; but couldn’t operate a stick-shift car. Can no longer ascend stairs.

For those with more severe HSP, a rubber bladder is surgically implanted the spinal column. It exudes into the nerves controlling the legs gamma-aminobutyric acid (GABA). This is the chemical missing in the affected motor neurons. GABA is an important (well, crucial) suppressor of nerve over-activity, hyper-excitability. When deficient, nerves fire too easily, causing all sorts of end-of-nerve anomalies.

My case of HSP is not bad enough to prompt the intense surgical procedure of implanting the rubber bladder in my spinal cord, thankfully. My case does not seem to be advancing, thankfully.

Here’s where blarcamesine will come into play with probably all forms of both HSP and PLS. In virtually all cases, neurons are lacking proper, effective concentrations of GABA; hence the over-excitability of the nerves. It was noted in the early findings of blarcamesine against Rett syndrome, in the early safety/tolerability study, that levels of GABA were dramatically increased, normalized by the drug; yielding increased normalization of motor neuron function.

Many years ago, there was a small study of blarcamesine included in the drinking water of lab rats with HSP genes. Like myself, they had reduced control and function of their spastic rear legs. Then, after they drank water with some blarcamesine in it, they rather promptly (in a week or two, as I recall) regained complete motor control of their leg muscles and twirled inside the exercise wheel like normal rats. Blarcamesine (Anavex 2-73) completely obviated the GABA deficiency, allowed normal walking. A cure, dare I say.

(Two years ago, my computer hard drive crashed; I lost the copy of that report; which I believe was in France. I’ve been unable to find it on the internet. If anyone can retrieve it, please post.)

A rare disease? Yes. “The HSP incidence rate in the United States is about 20,000 people.” But for many, very tragic, in the most severe cases:
https://sp-foundation.org/understanding-pls-hsp/hsp.html

When available, I’ll be one of the first to take blarcamesine, off-label, for my HSP. Will be rather expensive, my health insurance won’t pay for it. May have to liquidate a few AVXL shares, then.