Replies to post #342368 on Anavex Life Sciences Corp (AVXL)

[Bourbon_on_my_cornflakes]

The "whether it is precision medicine or not" debate reminds me of medieval theologians' "how many angels can dance on the head of a pin?" controversy.

Not sure who won that one.

Clearly a sign of those with too much time on their hands. Help us out with some real data, Dr. M.

At least the debate isn't about whether or not 273 is made of wood.

It matter not what it is called as long as the trial results show it is brilliantly effective in treating alz, Rett and PD...and soon others.

I don't think the FDA will care what it is called, if it works.

[WolfofMia]

If there is no selection on patients characteristics biomarkers, there is no precision medicine! There may be precision research, thoroughness etc.

At present there has been no selection and as you say because it appears all patients on average may benefit sufficiently well to achieve stat sig results without the need for very large trials. We don’t know for sure yet, but we will get insights to that when eventually we see about AVATAR and EXCELLENCE.

Well there may not have been patient selection, but you may still have precision medicine in the application end of the stick if information is discovered but not proved.

You may recruit without biomarker exemptions, I would start with the whole too, then break it down and disprove your theories as they may come.

But if in a P2 you find out there are gene exemptions which limit efficacy of your drug you may or may not prove this on a P3 if you don't need to, it is to your advantage to, but is your choice ultimately to do so or not.

If your drug works, stat sig efficacy and all, then you may not need to exclude patients in your P3, delaying your over all approval timing for extra information that may give you ultimately more knowledge of MOA. and disease pathology.

However with what you found out in P2 of potential gene limitation you may dose those patients with the expression or alternations of genes on the higher dose of the medicine. Action end of the sticks with findings from the P2, yet to be verified.

Would this classify as precision medicine?

[Amatuer17]

I agree with your argument.

See the definition of PM below. Is AVXL actually following any of those characteristics? A strong NO

1. They have not identified biomarkers that will differentiate patients
2. They have not used any characteristics in patients to develop patient specific medicine or dosing
3. They have not even designed the trial to select patients based on individual characteristics or biomarkers
4. The drug is being developed as a single dose for each and every patient.

So where is the precision part of drug development and trial. It is in M’s mind and in all his decks.

The definition of precision medicine is
“ precision medicine is "an emerging approach for disease treatment and prevention that takes into account individual variability in genes, environment, and lifestyle for each person." This approach will allow doctors and researchers to predict more accurately which treatment and prevention strategies for a particular disease will work in which groups of people.
It is in contrast to a one-size-fits-all approach, in which disease treatment and prevention strategies are developed for the average person, with less consideration for the differences between individuals.”

[bas2020]

If there is no selection on patients characteristics biomarkers, there is no precision medicine!

Please provide a link that states "selection" is a requirement for precision medicine.

Simple identification of the various pertinent genetic characteristics seems quite sufficient for a precision or personal approach.