Rapamune / Rapamycin/ mTOR

I just realised that they said that >100 people have enrolled, and that 40 are done.

This presumably means that they have published preliminary positive subjective results while >60 are still not done with their treatments.

I’m not sure I’m okay with this approach, as it could influence how the last >60 people perceive the treatment.

 

I would suspect so. They are not just showing positive results to their participants but changes in specific measures on subjective outcomes measures. I suspect that could lead to altering answering behaviour and other biases.

 

100% true this is one of the things people criticised the PACE trial for.

I still look forward to their final results but view them with a dollop of extra skepticism. My view is the likely effect is modest, most trial participants will never hear about it. The study looks set to go on to stage 3 anyway, that's where the blinding and the placebo group will really reveal the truth.

 

It seems to have been picked up by various SoMe ME/CFS news platforms, so I wouldn’t be surprised if some of them see it.

 

Rapamycin Improves PROMs in Patients With ME/CFS in Early Trial

Key Takeaways

• Rapamycin administration improved PROMs in ME/CFS patients, showing significant enhancements in fatigue, sleep, and orthostatic intolerance over 3 months.
• The study involved 40 participants, with rapamycin doses up to 6 mg weekly, demonstrating statistically significant improvements in multiple health scales.
• Findings suggest rapamycin's potential in ME/CFS treatment, possibly linked to its autophagy-promoting and mTOR-inhibiting properties.
• Further detailed data and subgroup analyses are expected to provide deeper insights into rapamycin's effects on ME/CFS.

 

From the article:

• BAS scores improved from 34.84 (standard deviation [SD], 3.13) to 42.26 (SD, 3.87; P <.001)
• SSS sleep scores improved from 7.35 (SD, 0.38) to 5.03 (SD, 0.58; P <.001)
• SSS PEM scores improved from 7.05 (SD, 0.39) to 3.77 (SD, 0.56; P <.001)
• SSS OI scores improved from 5.33 (SD, 0.46) to 3.50 (SD, 0.50; P <.001))
• MFI aggregated scores improved from 79.63 (SD, 1.43) to 72.26 (SD, 2.28; P <.001).

 

I haven't looked in detail but I get the impression that an opportunity was missed to do a dose response study. 100 subjects would be easily enough and we could have a very strong indicator of not only efficacy but optimal dose without placebo controls or even the need for blinding.

 

They mention that they are using a blood marker that responds to Rapamycin and if the marker increases and the patient improves it is related. Need to see the paper when it is written but I'm not holding my breath over the disaster of the Oxalacetate paper (my view, not others).

Given many patients don't tolerate larger doses when starting a medication they used 1mg titrating up to max 6mg as tolerated. So hopefully there will be different doses in the reporting.

From what I understand not all patients can tolerate the 6mg dose. Hopefully they report on how well tolerated the drug is.

Some patients had reactivated virus infection (confirmed with PCR) on the once weekly dose. Somehow that info seems to not be discussed in the multiple articles that have come out. Mouth ulcers were another issue which is a known side effect.

 

The improvements in scores don't look huge, do they?

Even a small improvement is a worthwhile result, obviously—what I mean is the drug as it's been used here doesn't appear to have made a big impression on whatever is driving ME/CFS symptoms. You'd expect a big shift if it did.

 

Sorry if I have missed the answer to this question but was the rapamycin there to increase of blunt mTor activity or sth else.
Interetsting to me since mTOR overactivity is involved in sarcoid and also suppressive of autophagy, which may be involved in my case as a Pompe carrier. May be grounds for cross condition consideration. (I appreciate that feedback mechanisms etc. may mean MTOR can act in more than one way).

 

Simmaron's Rapamycin ME/CFS Trial Moves Forward: The Goal - FDA Approval - Health Rising
Thanks. I read this review and it seems high ATG13 was the key issue. Possible subgroups?


Just for interest , I know this is not a sarcoid forum but:

https://www.google.co.uk/url?sa=t&r...usg=AOvVaw0muKvKmmkJiwkYC_Sp9m4o&opi=89978449

I think there are going to be things in common with sarcoid fatigue and Pompe sufferers also report a credible version of Exintol/PEM which sounds like sth beyond muscle weakness.