Ok, A Potential Big Negative (Or Not)
I’ve pondered this, that the base cause of Rett syndrome is genetic, an aberration in the genome the little girls inherit from their parents’ gametes.
Therefore, there can be no normal homeostasis to “restore.” None there to begin with.
In most Alzheimer’s cases the person was in general neurological health in earlier years, when organelles in the neurons functioned normally. Waste-clearing enzymes were synthesized and they did their job. But, with age, endoplasmic reticula began to break away from mitochondria, and Alzheimer’s symptoms set in.
Unfortunately, in Rett, there is no turning back to a better time, when things were working normally. Rett is a de novo (first time, no ancestral occurrence) mutation. In Rett, the production of a specific protein (MECP2) is genetically disrupted, complicating normal brain development.
How, then, might the known Anavex 2-73 sigma-1 receptor agonist work to fix this broken-from-the-start arrangement? I don’t see how this genetic problem can be fixed by restoring the mitochondrial/endoplasmic reticular connection. That works wonderfully in Alzheimer’s disease. But for Rett, there must be some other, yet undisclosed mechanism of action. I have no idea what that might be.
Not that my ignorance of the matter has any application, but it might be that Anavex 2-73 has a reduced chance of effectively treating girls with Rett syndrome; that, perhaps, it will provide only small or minimal benefits. Could up-coming Rett trial results turn out negative or meager? If so, what would be the impact on the future of Anavex Life Sciences Corp?
It could be imagined that a Rett trial failure would portend consequent Parkinson’s and Alzheimer’s failures. End of company. Has Missling bet the company’s future on a tiny population of diseased girls, for which the drugs might not work? How so?
One of two possibilities. The quick Rett trial (just 12 weeks) is a bet that results will be good, pushing things along for the other two, much more significant trials. A corporate gamble, perhaps, for a quick return?
Or, do the experts in the Anavex labs have information of yet-undisclosed new Anavex mechanisms of action that actually fix certain genetic errors? Ponder the implications of that.
Have Anavex scientists dosed some genetically deformed or deficient lab rats with Anavex 2-73 and found they then took up normal health or physiological function, by some new mechanism of action?
Frankly, I can’t see Missling and Company heading into an early, company-affirming clinical trial for which they haven’t solid understandings of just how it will turn out.
The science behind Anavex’s Rett therapy might be really big and new; portending a whole body of new applications for all sorts of genetic diseases. The Anavex story may turn out bigger than we presently imagine.
The Rett trial; bust or boom. I think, the latter.
