And, others, too.
Actually, for some time I've had just the slightest knowledge of ALS, encountered when I dug for information on the CNS disease that affects me, a form of Hereditary Spastic Paraplegia (HSP).
Fortunately, my HSP is "uncomplicated," meaning it's not severe. There are a multitude of HSP genotypes, and I've never had a genome assay to determine which form causes my HSP. For me (and most with HSP), the primary symptom is spasticity, particularly in my legs. They are continually stiff. To ambulate I require a wheeled walker, which I control with ease. Can no longer walk up a staircase, as I can only shuffle my legs along the ground. The spasticity (for me) causes no pain; just the ambulation difficulty and frustration.
But HSP has caused urinary retention. I've consequently had to have a suprapubic catheter surgically installed.
A treatment for HSP, expensive, often with complications, and not always so successful, is the implanting in the spinal column of a rubber bladder-like reservoir that mechanically expresses into the spinal column gamma-aminobutyric acid, GABA, which is able to turn down the chronic (always-acting) excitability of the long motor nerves that control muscles in my legs. For some, this brings certain levels of relief, but the bladder must be re-filled with a GABA solution by injection. Metering out precise amounts of GABA can be problematic. Because of all of that, I've learned to live with my HSP as it otherwise presents itself.
A number of years ago I discovered an obscure paper, I believe from France (written in English) where a researcher put blarcamesine in the drinking water of rats that had HSP genes. As in humans with HSP, the rats had poor control of their rear or lower appendages. The back legs were typically spastic.
But after drinking water with included blarcamesine, the rats in a short period of time gained full, normalized control of their legs; could spin themselves normally on a rat-cage wheel. Blarcamesine (then with a different name) was able to restore normalized GABA levels in the spinal column and motor-control (muscle controlling) nerves functioned normally.
Most unfortunately, three years ago the hard drive holding this file went out; I was not able to retrieve the document. I've searched hither and yon on the Internet, but have been unable to find it. For the HSP community, this report was extremely important; one of the only real hopes for successful treatment.
My personal goal is this. When blarcamesine is approved for Rett or some other however obscure CNS disease, my AVXL shares will have appreciated wonderfully. I'll sell a few so I can buy a prescription of blarcamesine for my off-label use, for my HSP. I'm confident that my neurologist will agree with this ploy. Of course, my health insurance company would not, so I'll have to pay, out of pocket, full price for the blarcamesine.
The significance of all of this, beyond HSP? Simple. Eventually, blarcamesine may be therapeutic, either by itself, or as an adjunct drug for the treatment of any number of CNS diseases. Right now, the foci are on just Rett syndrome, Parkinson's disease dementia, and Alzheimer's. Soon FTD, frontotemporal dementia, will be tested with Anavex 3-71 (as I understand it). Presently, we've discussed Anavex drug treatments or prevention of autism spectrum disorder.
Simply, Anavex has the potential of treating or preventing a large number of CNS and other diseases. I've refrained from projecting any of this (until here) for some time. Just as some readers will with this posting, many would have regarded my laying out the several (well many) other diseases that Anavex Life Sciences Corp might potentially treat as only "magic bean" thinking.
For the present, each reader will discern and decide for himself or herself. Later, clinical trials will decide the matter. There is a great deal of supporting, guiding evidence that will prompt new trials with other CNS diseases.
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