Question

I know that you have answered questions about the failure of people in the UK to have access to Ampligen – a drug that has immunomodulatory actions and is used to treat ME/CFS by some doctors in the USA. However, I’ve been told that the drug regulatory authority in America (the FDA) has now approved its use in treating Long Covid. So does this mean that doctors in the UK can now start prescribing Ampligen?

Answer

The situation here in the UK regarding Ampligen has not changed since we last wrote about it and is very unlikely to do so in the foreseeable future. As you are probably aware, the NICE Guideline Committee reviewed all the evidence for treatments that have been evaluated for ME/CFS in clinical trials. It concluded that there wasn't the necessary evidence on efficacy and safety to recommend the use of this very expensive drug here in the UK.

The FDA in America has approved an application by AIM Immunotech to evaluate the use of Ampligen in Long Covid in a phase 2 clinical trial starting in March 2023. If successful it would then move to a phase 3 clinical trial and if those results were positive there might be renewed interest in carrying out a clinical trial to evaluate its use in ME/CFS. But this does not mean that the FDA has approved Ampligen for either Long Covid or ME/CFS in the USA at this time. We need to wait on those trial results first, and then regulatory authorities including NICE would still need to approve its use here in the UK before it can be prescribed.

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 yet been established. The bottom line here is that despite all the hype that often accompanies Ampligen, there isn't sufficient evidence at present to conclude that this very experimental drug is a safe and effective treatment for general use in ME/CFS. Ampligen is not therefore a drug that is going to be prescribed in the UK on the NHS, and we think it’s very unlikely that you will find a private doctor who is willing to prescribe it.

Background

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

In March 2012, the results from a phase-3 prospective, double-blind, randomised, placebo-controlled trial compared twice weekly IV rintatolimod (Ampligen) versus placebo. It 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.

In December 2012, 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.

However, in October 2022, AIM announced that the FDA had given approval to “proceed to initiate a Phase 2 study evaluating its investigational drug, Ampligen, as a therapeutic for patients with post-COVID conditions.” Thomas K. Equels, MS JD, Chief Executive Officer of AIM went on to say:

“We are very pleased to be able to proceed with our Phase 2 study and are prepared to advance Ampligen as a potential therapeutic for the treatment of myalgic encephalomyelitis/chronic fatigue (ME/CFS)-like post-COVID conditions, an exploding area of significant unmet medical need,

“Post-COVID-19 Disabling Fatigue, along with other debilitating post-COVID conditions such as ‘Brain Fog’, continue to affect tens of millions of people worldwide…

“…we believe Ampligen has the potential to be an effective treatment option and an important solution for patients and physicians… We expect to commence enrollment in early 2023.”