Rapamycin therapy inhibits mTOR and reduces autophagy disruption. We believe that a subset of patients may have chronic mTOR activation that can lead to the symptoms of ME/CFS. By taking rapamycin, the mTOR inhibitor, we hope that these people may see a significant reduction in symptoms.
Simmaron Research
Extracts
Until now, the heterogeneity of the patient population and the lack of a reliable biomarker to diagnose and prognose ME/CFS has thwarted clinical trials for both non-approved FDA drugs as well as repurposed agents.
We have recently identified elevated levels of inactive ATG-13 as a testable and targetable pathway for treating symptoms of post-exertional malaise.
We will track symptoms and autophagy markers in this study.
Our publication on elevated ATG13 showed that a significant number of ME/CFS patients display serological evidence of autophagy disruption. We have shown that this deficit in autophagy is due to the chronic activation of mTOR. Without properly functioning autophagy, there is significant cellular stress, immune activation, and not enough energy for the cell to do well.
Rapamycin is an mTOR inhibitor. It is an FDA approved drug that was initially developed to protect patients during a kidney transplant. It has a well understood safety profile. This study will track autophagy markers and ME/CFS symptoms in patients who are treated with low-dose rapamycin by participating clinicians.
MEA Comment
Dr Charles Shepherd, Honorary Medical Adviser to the ME Association says:
Rapamycin is a rather unusual drug to be assessed in a pilot study (which may then progress to a proper clinical trial) for people with ME/CFS.
In very simple terms the drug is supposed to be able to block cells from multiplying and growing. It has therefore been used to prevent the immune system rejecting a donor kidney during the transplant process. Some doctors also claim that rapamycin can slow down the ageing process but the evidence is not very convincing.
Rapamycin has a number of potential side-effects – including weakening the immune system, anaemia, constipation or diarrhoea, fatigue, raising blood cholesterol and blood pressure, swollen hands and feet – which are clearly problematic if you have ME/CFS. More serious side-effects, although unusual, that have been reported include blood clots and strokes.
At present, I am not aware of any anecdotal evidence to indicate that rapamycin is a safe and effective drug to treat ME/CFS.
However, the pilot study is based on the possibility that a low dose of rapamycin could correct a chemical abnormality (ie elevated serum levels of a protein called pATG 13) that has been found in some preliminary research in a subset of people with ME/CFS. This research is described in more detail in the announcement from Simmaron Research.
In our current state of knowledge, I don't think rapamycin is a drug that should be used or prescribed outside a research setting and it is not something that a doctor here in the UK would therefore be willing to prescribe on the NHS.
Checking on Google it can, however, be purchased over the internet. This is not a route that I would recommend for a drug where we currently have no firm information on both safety and efficacy.
We will comment again when the results from the pilot study are published.
Useful review article in rapamycin below:
Dr Charles Shepherd,
Trustee and Hon. Medical Adviser to the ME Association.
Member of the 2018-2021 NICE Guideline Committee.
Member of the 2002 Independent Working Group on ME/CFS.
Dr Charles Shepherd comments
Rapamycin is a rather unusual drug to be assessed in a pilot study (which may then progress to a proper clinical trial) for people with ME/CFS.
In very simple terms the drug is supposed to be able to block cells from multiplying and growing. It has therefore been used to prevent the immune system rejecting a donor kidney during the transplant process. Some doctors also claim that rapamycin can slow down the ageing process but the evidence is not very convincing.
Rapamycin has a number of potential side-effects – including weakening the immune system, anaemia, constipation or diarrhoea, fatigue, raising blood cholesterol and blood pressure, swollen hands and feet – which are clearly problematic if you have ME/CFS. More serious side-effects, although unusual, that have been reported include blood clots and strokes.
At present, I am not aware of any anecdotal evidence to indicate that rapamycin is a safe and effective drug to treat ME/CFS.
However, the pilot study is based on the possibility that a low dose of rapamycin could correct a chemical abnormality (ie elevated serum levels of a protein called pATG 13) that has been found in some preliminary research in a subset of people with ME/CFS. This research is described in more detail in the announcement from Simmaron Research.
In our current state of knowledge I don't think rapamycin is a drug that should be used or prescribed outside a research setting and it is not something that a doctor here in the UK would therefore be willing to prescribe on the NHS.
Checking on google it can, however, be purchased over the internet. This is not a route that I would recommend for a drug where we currently have no firm information on both safety and efficacy.
We will comment again when the results from the pilot study are published.
Useful review article in rapamycin: