Ampligen for Chronic Fatigue Syndrome

Still Not FDA Approved

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What is Ampligen?

Ampligen (rintatolimod) in an experimental anti-viral, immune-system modulating drug that's been in the works for more than 30 years. It's been studied as a possible treatment for multiple conditions, including chronic fatigue syndrome (CFS or ME/CFS), HIV/AIDS, certain types of cancer, both avian flu and swine flu (H1N1), and the Ebola virus.

In 2009 and again in 2013, the FDA rejected Ampligen as an ME/CFS treatment.

It would have been the first approved treatment for this condition as well as providing Ampligen's entrance into the marketplace. In spite of these failures, the manufacturer continues to move ahead to get this drug approved.

How Does Ampligen Work?

Ampligen is believed to work by jump-starting your body's natural anti-viral pathway and regulating levels of RNase L (a substance in your cells that attacks viruses), which can be high in people with ME/CFS. It's also been shown to inhibit tumor-cell growth.

Ampligen doesn't appear to suppress the immune system. Instead, it's believed to change the way your immune system works.

Ampligen for Chronic Fatigue Syndrome

In clinical trials, Ampligen has been shown to improve cognition, exercise tolerance, neuropsychological health and overall function in people with ME/CFS; decrease activity of HHV-6 (a virus believed to be linked to ME/CFS); and decrease RNase L activity.

According to Ampligen's manufacturer, Hemispherx Biopharma Inc., more than 40,000 doses of Ampligen were given to about 500 patients in clinical trials at more than 20 U.S. clinics. One of those clinics, the Hunter-Hopkins Center, says 80% of its patients improved on Ampligen, and 50% improved significantly.

Because Ampligen isn't currently approved for any use in the U.S., only clinics with special permission can prescribe Ampligen. It's also incredibly expensive -- as in more than $40,000 a year! And no, your insurance won't cover it, because it's experimental.

Ampligen also remains unapproved in the European Union, but some patients there may be able to get it through what's called an "early access program."

Ampligen Dosage

Ampligen is administered intravenously (I.V.). In trials and under conditional permits by the FDA, patients typically have received 400mg of the drug twice a week. Hunter-Hopkins recommends at least 12 months of therapy, and 18 months for the severely ill.

Ampligen Side Effects

Like any drug, Ampligen can cause unwanted side effects. Possible side effects include:

  • Mild flushing,
  • Tightness of the chest,
  • Rapid heartbeat,
  • Anxiety,
  • Shortness of breath,
  • Feeling hot,
  • Sweating,
  • Nausea,
  • Liver enzyme level changes,
  • Diarrhea,
  • Itching,
  • Low blood pressure,
  • Rash,
  • Arrhythmias,
  • Low white blood cell count,
  • Dizziness,
  • Confusion.

Some patients have flu-like symptoms for a few hours after receiving a dose of the drug. The manufacturer says these effects typically went away after several months of treatment.

Sources:

Smith ME, et al. Annals of internal medicine. 2015 Jun 16;162(12):841-50. Treatment of myalgic encephalomyelitis/chronic fatigue syndrome: a systematic review for a National Institutes of Health Pathways to Prevention Workshop.

Strayer DR, et al. PLoS One. 2012;7(3):e31334. A double-blind, placebo-controlled, randomized, clinical trial of the TLR-3 agonist rintatolimod in severe cases of chronic fatigue syndrome.

Strayer DR, et al. Clinical infectious diseases. 1994 Jan;18 Suppl 1:S88-95. A controlled clinical trial with a specifically configured RNA drug, poly(I).poly(C12U), in chronic fatigue syndrome.

Suhadolnik RJ, et al. In vivo. 1994 Jul-Aug;8(4):599-604. Changes in the 2-5A sythetase/RNase L antiviral pathway in a controlled clinical trial with poly(I)-poly(C12U) in chronic fatigue syndrome.

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