Amx035

[nunhead_man]

Do any warriors here reckon this is worth a punt?

AMX0035 is a proprietary, oral combination of two drugs already in use, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA). PB is prescribed under the brand name Buphenyl to treat urea cycle disorders. It acts as a scavenger to facilitate the excretion of excess nitrogen. Interest in repurposing PB to treat neurodegenerative diseases stems from its action as a chemical chaperone, which inhibits endoplasmic reticulum stress responses and neuronal cell death induced by accumulation of misfolded or mutant proteins (Kubota et al., 2006). PB also epigenetically regulates gene expression by inhibiting histone deacetylase

https://www.alzforum.org/therapeutics/amx0035

I know I can get TUDCA over-the-counter but sodium phenylbutyrate (PB) needs a prescription it seems – and I would need to work out a sensible dose

[Ellie]

Clinical trial: AMX0035 twice daily--a combination therapeutic including 3 gram of Phenylbutyrate and 1g TUDCA.

You could try getting PB prescribed “off-label" by a Doctor. The Social Medwork sells it (deep pockets needed) and I daresay it’s available cheaper from less well-known vendors (caveat emptor)

[Lynne K]

I wouldn’t want to go it alone trying drugs. I’d want to be on a monitored double blind trial. We need to have our symptoms tracked before starting and throughout a trial, with half or whatever the scientists decide is a efficacious % participants on a placebo. Lynne

[nunhead_man]

Good morning,

Thank you Ellie – I might just try this dose to see if it has any effect although of course I won't know as I'm quite slow anyway but I am tempted.

And thank you Lynne – my scientific hat understands what you mean, especially in the light of what happened with the riluzole where as you may know the trial was shorter than might otherwise have been undertaken because of its positive effect and later research now suggests that it had more of an effect then was originally shown.

However, my personal hat says that anything I can grab that might slow or halt my progression would be something that I would welcome and I don't see why I should wait for big Pharma to get his act together and find the research money to do the work that I need for that purpose.

The analysis that is shown in the one in 300 blog (Lee Millard) points to just these kinds of challenges in finding medicines for a syndrome that is in its current diagnostic state

[Lynne K]

Thanks for the info Andy and good luck, Lynne

[Onein300]

Good morning,

Thank you Ellie – I might just try this dose to see if it has any effect although of course I won't know as I'm quite slow anyway but I am tempted.

And thank you Lynne – my scientific hat understands what you mean, especially in the light of what happened with the riluzole where as you may know the trial was shorter than might otherwise have been undertaken because of its positive effect and later research now suggests that it had more of an effect then was originally shown.

However, my personal hat says that anything I can grab that might slow or halt my progression would be something that I would welcome and I don't see why I should wait for big Pharma to get his act together and find the research money to do the work that I need for that purpose.

The analysis that is shown in the one in 300 blog (Lee Millard) points to just these kinds of challenges in finding medicines for a syndrome that is in its current diagnostic state

Andy, the rule we should perhaps aspire to is:

If it’s cheap, does no harm, try it if you want.

Sadly I have seen this again and again with supplements. People jump on any sound bite. Trehalose is the current one. The trial is IV but people are shovelling it in orally! More likely to cause side effects etc, not reach target etc etc.

My interpretation of amx0035 data released is very simple:

Although It appears to slow progression it is not a game changer. All participants appear relentless on their deterioration. We are looking for progression stoppers, ie 100% slope flattening.

It’s not at all clear if it will be approved by FDA in the near future. Based on the data published I can’t see it being approved here in UK. The MHRA have a higher bar to jump re cost/effectiveness ratio with NICE.

If I was interested (which I am not) I would just try Tudca, but do please check with your doc.

Lee

[Banned]

Hi Andy,

You first need to know what the problem is with your neurones.

Are they dying prematurely?

Is the signal junction degraded?

Is the protein manufacturer knackered?

Have the apparatus that services the neurones seen better days?

Our bodies are nothing more than incredibly sophisticated machines and like with all machines, they fall apart into disrepair and the only way to fix it is to know what has gone wrong. A gene test may provide the answer.

[jd58]

Yes thats what mnd does to neurones

[House44]

Is any of you taking this and do u find it has had any effects?

[nunhead_man]

Good afternoon

Is any of you taking this and do u find it has had any effects?

No I'm not as yet because I cannot find a way of doing it.

The bit of this mixture that is prescription based is in fact eyedrops as far as I can see so I can't quite work out how to put together a sensible dose as represented by the trial.

Although as Lee says above, perhaps I'm wasting my energy and it will be really good to have something that flattened the curve by one hundred percent

[matthew55]

When they come up with a drug that can repair my dead tounge and voice box I will take notice. 😉😁x