The study paper’s title begins with: “Low Dose Rapamycin Alleviates Clinical Symptoms
of Fatigue and PEM in ME/CFS Patients […]”. It sounds good… the title is saying that the treatment reduces symptoms.
But if you start skimming through the pre-print paper, you will see that only 40 out of 86 patients actually completed the study. Unfortunately, the study didn’t go so well and the number of people dropping out is much higher than what’s ideal.
Now suppose that there is a treatment that does nothing and that patients have a roughly 50/50 chance of getting better/worse over time. If the patients who get worse all drop out of the study, then the final cohort will have an overrepresentation of people who got ‘better’. This is why I would not draw conclusions from this study design (which is not ideal) when the dropout rate is so high.
Possible reasons why the dropout rate was so high
The preprint states that study participants had to pay to stay in the trial. While Simmaron Research’s charitable mission is to conduct research, it seems that they didn’t fund higher-quality research despite raising six figures every year since 2011. (Note: charities have overhead and have to find a way to pull in enough money to survive.)
Importantly, discontinuation from the study was most commonly attributed to financial barriers as this pilot trial did not cover the cost of the study drug or safety laboratory tests.
The pre-print also explains that patients sometimes left the trial if they weren’t getting better:
A secondary reason for discontinuation was a lack of perceived clinical benefit or a clinical decision to initiate a different therapy that may or may not interact with the study protocol.
Twitter thread by one of the paper’s authors
Patients have had bad experiences with antibiotics
Survey data that I collected show that patients (LC, MECFS, and vax) are more likely to report bad experiences with antibiotics than other treatments.
Caveats:
- Survey data is unreliable in certain ways. (I don’t want to type out a nuanced deep dive on that though.)
- What is true for antibiotics in general doesn’t necessarily apply to rapamycain.
- While people did try rapamycin (or other macrolides), rapamycin wasn’t very popular among the survey respondents.
Sources:
- see slide 19 https://longhaulwiki.com/resources/assets/What-worked-Feb-16-2023.pdf (but also see slide 16)
- This Odysee video goes over some of the Treatment Outcomes Survey.
- Information on the successor survey (Patient Experiences Survey) is here.
Survey data suggests actual response rates are in the 1-20% range (maybe around 2%), and that human trials should ideally have many participants and a control group (because there is very little signal in the survey results and a lot of noise from reporting biases).