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frrol

05/13/20 12:11 PM

#250548 RE: falconer66a #250541

And yet most people with AD given it in a small uncontrolled trial did not show a stat-sig response, and a significant proportion withdrew consent to continue. And the millions of AD sufferers already being dosed with a lab-verified S1 agonist have not been "cured" nor deemed effectively tested.

Let's take it slowly. This is risky business and a big, controlled trial is what separates the real therapies from the amazing, can't-lose, the-science-is-amazing ones that heartbreakingly fizzle. And the latter greatly outnumber the former.

We have PDD results coming up that should be informative. It's exciting and hopefully we'll have positive signal.

jimmy_mcyoloswag

05/13/20 12:14 PM

#250549 RE: falconer66a #250541

Sigma1 receptor activation apparently improves mitochondrial function via modulating activity at the Mitochondria-ER „docking site“ (MAM) through a host of signalling cascades related to ER-stress, inflammation, autophagy and mitochondrial homeostasis. One in particular: Calcium

The MAM:
https://www.sciencedirect.com/science/article/pii/S0005272813001850

S1r and the MAM:
https://www.sciencedirect.com/science/article/pii/S134786131400022X

Sounds to me like S1r are the mechanics at a space dock where mitochondria swing by for service..

abew4me

05/13/20 2:28 PM

#250574 RE: falconer66a #250541

If AVXL's A2-73 results are minimal but are enough to receive some kind of approval, can they use their Rare Orphan Drug Designation to fast-track the 3-71?