Medical Matters > Treatment: Ampligen

ME Essential Summer 2022


I have read a lot of information about the use of a drug called Ampligen on the internet. While it is being given to people with ME in America it does not appear to be available here in the UK

My understanding is that Ampligen is being used by a number of doctors who specialise in ME in America and that some people with ME have gained considerable benefit.

When I asked my GP about Ampligen he knew nothing about it and did not want to even look at the information I took to the consultation, or investigate further. Even if it means paying privately I would like to give Ampligen a try. I am prepared to accept that it may not work and that it does have side-effects.


Ampligen is the trade name for a very expensive American drug that is claimed to have both antiviral and immuno-modulatory properties. As you say, benefits have been reported by a number of clinicians (mainly in America) who use this drug in selected ME/CFS patients.

There have also some small clinical trials in America. A phase-3 prospective, double-blind, randomised, placebo-controlled trial comparing twice weekly IV rintatolimod (Ampligen) versus placebo was conducted in 234 subjects with longstanding and debilitating ME/CFS at 12 sites by Strayer et al. Rintatolimod produced objective improvement in exercise tolerance and a reduction in ME/CFS-related concomitant medication usage as well as other secondary outcomes.

However, a US Federal Drugs Agency (FDA) advisory panel voted 8-5 against recommending approval for the drug for general use in ME/CFS in America. The FDA then approved what is called a compassionate care programme for Ampligen which allowed treatment of up to 100 ME/CFS patients at any one time at approved clinical infusion therapy sites.

Ampligen may be of use in a sub-group of people with ME/CFS – but identifying which people might fall into this sub-group has not been established. The bottom line here is that despite all the hype that often accompanies Ampligen, there isn't sufficient evidence to conclude that this very experimental drug is a safe and effective treatment for general use in ME/CFS. Consequently, the NICE guideline committee decided that it could not be recommended as a treatment for ME/CFS in the new NICE guideline.

Ampligen is not therefore a drug that is going to be prescribed here in the UK on the NHS.  And I think it’s very unlikely that you will find a private doctor who is willing to prescribe it in our current state of knowledge.


The improvement observed represents approximately twice the minimum considered medically significant by regulatory agencies. The rintatolimod cohort vs. placebo also reduced dependence on drugs commonly used by patients in an attempt to alleviate the symptoms of CFS/ME (p = 0.048). Placebo subjects crossed-over to receive rintatolimod demonstrated an intra-patient improvement in ET performance at 24 weeks of 39% (p = 0.04). Rintatolimod at 400 mg twice weekly was generally well-tolerated.


Rintatolimod produced objective improvement in ET and a reduction in CFS/ME related concomitant medication usage as well as other secondary outcomes.

D R Strayer et al. A double-blind, placebo-controlled, randomized, clinical trial of the TLR-3 agonist rintatolimod in severe cases of chronic fatigue syndrome | March 2012


Medical Matters is for information purposes only. The answers provided by Dr Shepherd and the ME Association’s other expert advisers should not be construed as medical advice. We recommend that any information you deem relevant is discussed with your GP as soon as possible. It is important to obtain advice from a GP who is in charge of your clinical care, who knows you well, and who can consider other likely causes for symptoms. Seek personalised medical advice whenever a new symptom arises, or an existing symptom worsens. Don't assume that new or worsened symptoms are a result of having ME/CFS.

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