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Re: kevindenver post# 408739

Tuesday, 03/28/2023 2:51:34 PM

Tuesday, March 28, 2023 2:51:34 PM

Post# of 461425
It's An Insurance Thing.

Why go through the trouble of another phase one after a Rett Syndrome approval? [To get blarcamsine used for HSP.]

So many doctors prescribe off-label wouldn't than be more prudent?


Well, yes it would. When blarcamesine shows wonderful results with girls with Rett, whose muscle control problems are then fixed, just have physicians prescribe blarcamesine off-label for patients with hereditary spastic paraplegia (HSP), even though it hasn't been directly approved for that rare disease yet. Makes sense.

But not dollars. No health insurance company is going to fork over tens of thousands of dollars for the off-label use of a drug not yet approved to treat an extremely severe central nervous system (CNS) disease, such as complicated HSP.

With my "uncomplicated," mild case of HSP, where I can get around on my walker and have no other progressing symptoms, I'm certain that once I make my case to my neurologist, with a 12-page document I'll write, he will prescribe blarcamesine for me. Wonderful. Very likely I'll be able to walk again, and go up and down stairs.

But my health insurance won't cover the costs. I'll have to sell a few of my AVXL shares and make the payment myself. Waiting for the opportunity for all of this to take place.

Now here's a new scenario; have never seen it presented before on this board (or anywhere else). If it can happen, a really Big Thing.

In most of the CNS debilities blarcamesine is being targeted at, they are not natally present; existing at birth. They come on much later in life. My form of HSP has its onset in late mid-life, in the fifties or sixties. Alzeheimer's and Parksinson's diseases, in most cases, are of geriatric onset. Now, in all of these, here's the potential Big Thing with blarcamesine.

In all three of these diseases, blarcamesine should restore normal neuron functions. Brains and nerves work normally, as they did in more youthful periods. It took some time for the three CNS disorders to work their pathological mischief. Could it be that blarcamesine is a CNS nerve re-set agent, where not only are neuron functions restored to a youthful state but that that restored youthfulness is retained?

Could it be that after a lengthy therapeutic period; say, two or three years, nerves have been permanently restored to their youthful state and blarcamesine therapy would no longer be needed? Very possible, I believe.

Of course, I'm betting that Dr. Missling already knows if this permanent neuron function re-set actually happens. His back-lab research people have already done dose-terminating studies in transgenic murines. The lab rats or mice suffered from some human CNS-disease genes got successfully treated with blarcamesine; which after a period of therapeutic success was then terminated. What happened then, when the blarcamesine was stopped? Do you suppose they rather quickly went back to their CNS disease states? Or, did the restoration of neuron physiology chronically (over time) persist?

In time, when the fullness of Anavex science is known and released, we'll find out. By that time, I hope to be walking around normally again; and I, like the rats, will be able to terminate my successful blarcamesine HSP therapy.
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