Ampligen I: Effectiveness

Ampligen (AMPlIfied GENetic Activity) is

a double-stranded RNA molecule developed in the 1960’s by Merck and Co to reduce tumor formation. Effective in the lab but too toxic for use Ampligen languished until Dr. William Carter, working with other Johns Hopkins University researchers in the 1970s, was able to reduce its toxicity.

Ampligen’s reported mode of action – bolstering the immune system against a variety of pathogens – is unusual and has lead to skepticism in the medical community; few drugs are effective against more than one type of pathogen.In April of 2007, however, Hemispherx Biopharma, Ampligen’s producer, reported that Ampligen works by activating pathogen detecting receptors on the surface of the cells called TLR-3’s. Ampligen is the first of a new class of drugs that attempt to effect TLR receptor activity with several drugs expected to vie for FDA approval in the near future.

In 2009 Ampligen was undergoing trials in several diseases, including chronic fatigue syndrome (ME/CFS), multidrug resistant HIV/AIDS and hepatitis C.

Hemispherx Biopharma is a small biopharmaceutical company specializing in RNA nucleic acids for use in viral disorders and other debilitating illnesses. It has four products in its portfolio, one of which, Alferon-N, is approved for use in sexually transmitted diseases. Oragen, an oral form of Ampligen is being evaluated.

Ampligen May Work in Chronic Fatigue Syndrome (ME/CFS) Because

it modulates the activity of a part of the innate immune defense (interferon pathways/RNase L enzyme) that some studies suggest is disrupted in chronic fatigue syndrome (ME/CFS). It does this through its ability to mimic the structure of viral proteins which normally stimulate the body’s immune defenses. Some chronic fatigue syndrome (ME/CFS) patients have high levels of an unusual type of a molecule which appears to disrupt the innate immune response. Ampligen appears to be able to restabilize the immune response in at least some of these patients.

Hemispherx Biopharma recently reported that Ampligen stimulates the activity of the ‘toll-like’ receptors on the outer part of the cell that play an important role in the early recognition of pathogens. Some but not all studies suggest that rates of opportunistic infections are increased in chronic fatigue syndrome (ME/CFS) patients. Ampligen’s broad anti-pathogenic properties may be able to reduce the activity of these pathogens in some patients.

The Ampligen Series on Phoenix Rising

  • Ampligen – What it is, how it works, what doctors say, etc.
  • Twisted History – Ampligens long and tangled road with ME/CFS
  • Dr. Lapp Interview – Kelvin Lord interviews long-time Ampligen provider, Dr. Charles Lapp, on Ampligen and other treatment options
  • The Ampligen Blogs – Follow Kelvin Lord as he documented his year-long experience with Ampligen

Chronic Fatigue Syndrome (ME/CFS) Patients Report –

Ampligen is clearly very effective in some patients; from Andrea Whittemore to Mary Schweitzer to Bob Miller to Kelvin Lord, stories abound of patients literally almost rising from the dead when being given Ampligen. While the optimism regarding the drug has become tempered over time and Ampligen is no longer spoken of in terms of a ‘cure’ Ampligen has nevertheless significantly helped some very ill patients…the only question about Ampligen is what percentage of the population it could help.

Chronic Fatigue Syndrome (ME/CFS) Studies: The first published Ampligen studies on chronic fatigue syndrome (ME/CFS) date back almost 15 years. A 1994 placebo-controlled, double-blinded study found significant increases in Karnovsky scores, increased performance in treadmill tests and improved cognition. A review poster at the 2007 IACFS/ME Conference indicated chronic fatigue syndrome (ME/CFS) patients (n=326) taking Ampligen received a 15% increase in endurance on a treadmill versus a placebo group. In 2012 Hemispherx completed a major study showing increased exercise tolerance, somewhat increased Karnovsky scores and increased vitality scores.

Chronic Fatigue Syndrome (ME/CFS) Doctor’s Report – A small group of doctors (Dr. Lapp, Peterson, Klimas, Podell, Bateman, Cheney) have been involved, on and off, in Ampligen treatment trials for the past 20 years.

In 1997 Dr. Lapp was very high on Ampligen citing a report showing that Ampligen worked ‘marvelously’ with 80% of patients displaying significant improvement and 50% recovering (!). Ten years later he was decided more subdued stating that while he’d had ‘some real successes’ with many patients getting better he did not consider it a ‘cure’. His website states that of 26 patients using it at his clinic 12 reported ‘significant improvement’ and six ‘some improvement’.

Dr . Bateman reported that she feels “confident that Ampligen improves the symptoms and function of some patients after weeks to months of use.” In the Sept. 2004 CFSAC meeting she noted that the majority of patients who received the drug demonstrated a clear improvement.

After working with Ampligen for decades Dr. Peterson reported that the drug is ‘quite effective”.

While the improvements seen in the studies are modest Dr. Bateman noted that “these are people who normally can’t do anything strenuous without getting sick, and the majority of patients experienced an improvement in their most difficult-to-reverse symptoms”. Dr. Klimas echoed that “This drug is quite beneficial for some patients, and the next step is to figure out how to predict which ones this can truly help.”

Dr. Teitelbaum does not appear to have participated in Ampligen treatment trials but feels that, given its high costs and potential side-effects, a multi-systemic approach is more desirable. He stated it was one of the last drugs he would recommend.

One report of high relapse rates after several years off the drug suggests that patients may have to periodically undergo cycles of treatment.

Who Might Benefit 

Given Ampligen’s high expense (reportedly $15,000-25,000 a year), an important question concerns which patients Ampligen will work in. Unfortunately neither the physicians in the clinical trials (Dr. Lapp, Bateman, Klimas) nor Dr. Carter (during a 2007 press conference) have been able to identify Ampligen receptive patients. Dr. Lapp has stated that ‘patients of all ages, severities and duration’ have benefited”

In 2011 Hemispherx joined with a biotech company called Chronix to develop a diagnostic test for ME/CFS using gene analysis methods it believes will also be able to highlight patients who do well on Ampligen.

Side Effects

Dr. Bateman reported no significant side effects in a 2004 trial. Side effects were reported to be low and did not significantly differ between the placebo and the Ampligen group in the 2007 IACFS/ME Conference report. Dr. De Meirleir reported that Ampligen is able to reregulate the RNase L enzyme system but may have negative effects in some patients through its upregulation of another immune enzyme, protein kinase R. The 2012 PLOS study reported that mild of moderate levels flu-like syndrome, chills, vasodilatation and shortness of breath were increased in some patients. In general the side effects appear to be low and certainly lower than in other immune modulating drugs.


Ampligen (400 mg.) is generally administered intravenously twice weekly for an hour. One of Dr. Peterson’s patients reports, however, that Dr. Peterson believes the correct dosage is variable with the standard dose (400 mg) often being too high and the appropriate dose being as low in some patients as 25 mg. Temporary flu-like symptoms are common. While six months may be enough to produce a significant response, Dr. Lapp recommends 12-18 months depending on the patient’s condition.

Infusion costs range from $1-2,000/month; $15-25,000 a year depending on dosage.

Recent History

Hemispherx appears to have gotten its financing and legal house in order with Dr. Carter reporting that all legal issues have been settled leaving the company free to focus on the drug itself. Ampligen has completed Phase II and III clinical trials. After having missed the deadlines in 2005 and earlier in 2007, Hemispherx filed its ‘New Drug Application’ (NDA) with the FDA in October, 2007. The FDA bounced back the application with questions in early 2008. Hemispherx replied and after both the drug and the company went under further reviews the FDA passed the drug onto the final review.

Studies indicated that Ampligen was more effective than other FDA approved drugs in increasing cardiovascular endurance but in 2009 the FDA declined to approve the drug citing the need for a variety of expensive studies which appeared to be well beyond Hemispherx’s ability to finance leading some commentators to predict the end of Ampligen’s road at the FDA. In 2012, however, Hemispherx received an extension on its application from the FDA.

Hemispherx’s 2012 study publication containing study data dating back almost a decade, suggested the company does not have the funds to comply with the FDA’s requests. It’s unclear where the best drug developed for ME/CFS stands now.

Share this!