Anavex Life Sciences Corp (AVXL)

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‘Biomarkers’ and therapeutic efficacies.

Understandably, a frequent topic has been ‘biomarkers,’ as they might indicate therapeutic efficacies of certain drugs, or the presence of actual disease conditions, etc.

What, actually, constitutes a ‘biomarker?’ What is a biomarker? Here’s where the FDA defines it: https://www.fda.gov/drugs/biomarker-qualification-program/about-biomarkers-and-qualification

Specifically:

“The Biomarkers, EndpointS and other Tools (BEST) glossary defines a biomarker as a defined characteristic that is measured as an indicator of normal biological processes, pathogenic processes, or responses to an exposure or intervention, including therapeutic interventions. Molecular, histologic, radiographic, or physiologic characteristics are types of biomarkers. A biomarker is not an assessment of how an individual feels, functions, or survives.

I find interesting the last sentence in the FDA’s definition—which I believe should be kept in mind regarding blarcamesine:
A biomarker is not an assessment of how an individual feels, functions, or survives.

Clearly, the the FDA separates how a biomarker is used in a clinical trial, compared to how patients in that trial actually responded to the drug being tested. No connection to how the patients felt, functioned, or survived after being on the drug.

What, then, is the function of either clinical drug tests, or the therapeutic administration of any drug? Aren’t drugs supposed to share two traits, a) to work safely, and b) to provide favorable therapeutic outcomes (making patients “feel better, function better, or survive better”)? Focusing solely on biomarkers simply avoids determining if patients got better. They are, as the FDA defined, only “Molecular, histologic, radiographic, or physiologic characteristics....”

Isn’t the presence of beta-amyloids in neurons or nerves a biomarker for Alzheimer’s? Except—lots of people have lots of amyloids in their brains, equivalent to others with profound Alzheimer’s; but have no Alzheimer’s symptoms. Simply, beta-amyloids are not a reliable or accurate predictor or trait of Alzheimer’s. In many with the disease, yes. But beta-amyloids don’t necessarily, in every case produce Alzheimer’s symptoms. Lots of people have lots of amyloids, but no Alzheimer’s (as posthumous histological examinations of brain tissues have revealed).

Personally, I’m not much interested in the clinical ‘biomarkers’ arbitrarily connected to blarcamesine therapy. I’m not much interested at all if the Anavex drug increases the synthesis of sigma-1 receptor proteins. If it does (as current research indicates), well and good. But the only things that really matter are safety and efficacy. Does blarcamesine administered to patients with accurately diagnosed CNS diseases work safely and reduce symptoms, or not? ‘Biomarkers’ are effectively irrelevant. Is the drug safe and effective, or not? Let actual therapeutic outcomes of the three on-going clinical trials of blarcamesine answer those two seminal questions.