Anavex Life Sciences Corp (AVXL)

[falconer66a]

Thinking Outside of the Constraining RCT Box

Well, thank you for posting this account of the medical professor fixing his alcoholism with baclofen. I was not aware of the fellow nor baclofen's ability to treat alcoholic dysfunction.

But, I'm not surprised by how the fellow figured it out.

He healed himself by taking baclofen, very off-label, after reading journal articles that led him to believe it would be effective. He experimented to find his proper dosage.

In general science, and in this case in particular, in medical science, there are a diversity of ways of looking at various problems and how they might be addressed. In conventional, modern science there is the "create an hypothesis and test it" approach. It's standard research procedure for the past century or so. Find a problem, figure out proposed solutions, and properly test them (which in medicine requires proper randomized controlled trials, RCTs). The standard stuff we know about here. Give some patients the real drug; other patients a starch pill that looks exactly like the drug pill. Two "arms" in the study, the placebo or control arm, with the fake but good-looking drug, and the experimental arm, where patients are getting the real drug. Patients in both arms, along with all of the medical professionals, can't detect or know which arm they are in. Results then aren't swayed by hope or belief; only by authentic drug-induced outcomes. This really works; it's the way drugs are required to be tested. Well and good.

But the good cardiology professor with a serious alcohol problem didn't restrain himself to RCT data. On his own, he went off and did things not included in RCT testing. He simply read everything he could find on his problem and found some data showing that baclofen might work. So, he experimented on his own, on his own body even; and found a solution. His findings aren't in any medical textbook or journal. It's categorically rejected by the medical community. "Nope. We can't consider that at all. No replicated double-blind studies to show that it actually works. Very bad, risky medicine."

The fellow was actually endangering his certification as a medical professional. "We can't have people like this doing actual medical research; a danger to themselves and to misinformed patients."

I've been there myself. Six years ago, because of the neurogenic bladder from my HSP, I had severe sepsis; a body-wide infection. I was a few hours from death; but a team of nephrologists caught the sepsis in time and I survived. But it appeared that my kidneys were failing and was told that as soon as my strength returned I'd need a transplanted kidney, or life-long dialysis. Not good.

But like the researcher mentioned in this post, I, too, didn't restrict myself to information from randomized controlled trials (at least those in the Western World). I found a study in India, of all places, showing that the taking of 4 or 5 ml of a dietary oil used in condiments in the East could save failing kidneys.

I bought the stuff, started taking it (a teaspoon in the morning and another at night), and at my next nephrology appointment the good doctor said something was wrong with my kidney tests. My kidneys were working better, not worse. Unheard of.

For six years I've been doing this; have reversed my chronic kidney disease from Stage 4 (soon to require transplant or dialysis) down to a continuing Stage 3. No transplant or dialysis needed. This was contrary to all of what American nephrology knows and believes from accepted RCTs.

Does any of this relate in any way to Anavex? Sure does. The science of the Anavex molecules, acting in neurons and as ligands of the sigma-1 receptor protein, is entirely new science (in the last decade). Publicly the depths of this new science are not completely known or revealed. To presume that the Anavex molecules might potentially treat only the three diseases presently being tested, Rett syndrome, Alzheimer's disease, and Parkinson's disease dementia is typically short-sighted; constrained by the mere three clinical trials for the three diseases.

Watch, in the next five to ten years a lot more diseases and conditions will be found amenable to successful blarcamesine therapy. Anavex, privately and quietly, is thinking far outside of the box containing the existing randomized controlled trials.