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Investigational Drugs
Ampligen, also known as poly I: poly C12U, is an immune modulator developed by Hemispherx Biopharma of Philadelphia, Pennsylvania. Ampligen activates the TLR3 receptors on respiratory epithelium, which is responsible for recognizing pathogens immediately, even those the body has not yet encountered, such as viruses and certain forms of cancer. Ampligen was first synthesized in the 1970s and has been proposed and tested as a treatment for many illnesses, primarily CFS and AIDS.



AMPLIGEN
(immune modulator)




Patients with CFS have severe ambulatory problems related to diminished uptake of oxygen, similar to patients with severe angina or chronic respiratory disease. A total of 5 open label and 3 double blinded placebo controlled trials have been completed, demonstrating that patients treated with Ampligen for 40 weeks or more, had improved exercise treadmill performance and their Karnofsky performance scores also improved significantly. 
In addition to Chronic Fatigue Syndrome, Hemispherx has also been studying Ampligen's use in myocarditis.  Studies in Belgium have shown that Ampligen can protect mice from CVB-3 induced myocarditis by 98% when administered at 20 mg/kg/day. Ampligen has also shown a reduction of CVB RNA in the heart, and the electrocardiogram in treated mice returned to normal. 
Pleconaril (capsid binding inhibitor)
Pleconaril Chemical Structure




Pleconaril is an experimental antiviral being developed by Schering-Plough. It is designed to prevent asthma exacerbations in asthmatic patients exposed to picornavirus respiratory infections.  Although the drug is delivered by nasal spray, pleconaril is also orally bioavailable, and active against viruses in the picornavirus family, including enteroviruses and rhinoviruses. 
Pleconaril binds to a hydrophobic pocket in VP-1, the major protein in picornaviruses. In enteroviruses, this prevents the virus from exposing its RNA, and in rhinoviruses, it also prevents the virus from attaching itself to the host cell.
Pleconaril was originally developed by Sanofi-Aventis, and then licensed to Viropharma in 1997. Viropharma tried to develop pleconaril for the treatment of acute enterovirus infections in infants and young children, but their application was denied by the FDA in 2001, citing safety concerns.
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