Rintatolimod
| File:Ampligen.jpg | |
| Clinical data | |
|---|---|
| Routes of administration | IV |
| Identifiers | |
| PubChem CID | |
| CompTox Dashboard (EPA) | |
| Chemical and physical data | |
| Formula | C28H40N9O25P3 |
| Molar mass | 995.584 g·mol−1 |
Ampligen, also known as polyI:polyC12U, is an experimental antiviral and immunomodulatory double stranded RNA drug developed by HEMISPHERx Biopharma, Inc. It is believed to be of use in the treatment of Chronic Fatigue Syndrome (CFS), a severely debilitating disease. Ampligen completed clinical trials phase III for CFS in 2004[1], and an extension of the interventional uncontrolled open trial under the cost recovery programme (Amp 511 )is currently recruiting [2]. A new drug application, or NDA, is in preparatory stages, and Hemispherx management has targeted late 2006 for its filing with the U.S. Food and Drug Administration, although the company has missed several target deadlines for an NDA filing in the past, including the end of 2005 and the third quarter of 2006 [3].
History
Mechanism Of Action
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The mechanism of action in relation to CFS is not entirely clear, but it is known to act on two important enzyme systems, one of which is the 2-5 Synthetase/RNase L anti-viral pathway. Research increasingly shows that the upregulation of this pathway is an important factor in the development of CFS [4]. The downregulation of this pathway leads to the immune system being better able to destroy viral RNA and accelerates the apoptosis of virally compromised cells[citation needed](quotation?). Ampligen must be received intravenously, but Hemispherx is researching an oral form of the drug [5]. It is generally administered twice weekly for periods of one year or greater.
Impact
Approximately 760 patients have received Ampligen as part of clinical trials in the US, representing about 75,000 doses. Some patients have reported a complete recovery, with others reporting clear and measurable improvements [6], while others have suffered relapses and serious side effects(quotation?)[citation needed]. More common benefits include improved cognitive skills, an increase in energy, and greater oxygen uptake. These improvements can be measured on the Karnofsky scale. Some studies suggest that Ampligen is most effective against a certain subset of CFS patients (quotation?), those with severe debilitation. Other studies indicate that Ampligen has a more significant effect on those who have suffered for 5 years or less, for reasons relating to activation of the 2,5 Rnase pathway (quotation?)[7].
Side Effects
Reported side effects include flushing, chills, fever, malaise, leukopenia, neutropenia and leukocytosis. A full list is available on the Hemispherx website [8]. The extent of these side effects are unknown, although Hemispherx claim they usually subside within 'several months'.
Controversy
Ampligen has been surrounded by controversy
Other Uses
Alternative potential uses of Ampligen include fighting Avian Flu, AIDS [9], Hepatitis B and Cancer, [10].