Journal of IiME Volume 4 Issue 1 www.investinme.org Ampligen® in Severely Debilitated CFS Patients Introduction Chronic fatigue syndrome (CFS) is a seriously debilitating disorder characterized by disabling fatigue and a combination of flu-like symptoms. The fatigue is not improved by bed rest and may be worsened by physical activity. The Centers for Disease Control (CDC) has identified CFS as an economically and emotionally devastating illness whose functional impairment can be equivalent to multiple sclerosis, heart disease, chronic obstructive pulmonary disease, or end-stage renal disease. The etiologic basis for CFS is unknown and may be multifactoral. With no approved drug therapy available, treatment is aimed at symptom relief and improved ambulatory function. Ampligen® [rintatolimod; poly(I) ·poly(C12,U)] is a double-stranded RNA compound in late-stage clinical development for treating CFS and a New Drug Application (NDA) has been filed with the US Food and Drug Administration (FDA). Exercise tolerance (ET) testing is an objective measurement of treatment efficacy and is accepted as a standard test for drugs ameliorating exertional fatigue. AMP-516, the key Phase 3 multi-center, doubleblind, placebo controlled trial, used ET as its primary endpoint in the evaluation of Ampligen® in the treatment of CFS through its action as a selective Toll-like receptor 3 (TLR3) agonist. TLRs are evolutionary preserved host defense systems which recognize various “microbial mimics”. TLR3 specifically recognizes double-stranded RNA, part of the replicative cycle of many pathogens including viruses. Given the likely multifactorial etiology of CFS, the rationale of the study was to determine whether chronic exposure to a selective TLR3 ligand would result in quantitatively and medically significant ambulatory/exercise improvements. As has been previously communicated, Hemispherx Biopharma received a completed response letter from the FDA regarding the NDA for the use of Ampligen® to treat CFS. In this letter, the FDA has recommended at least one additional clinical study which shows a convincing effect and confirms safety in the target population. Since receiving the letter, Hemispherx has instituted a new clinical study protocol to Invest in ME (Charity Nr. 1114035) Ampligen® in Severely Debilitated CFS Patients 1, 2 Article submitted by Nancy McGrory Richardson, Education and Outreach Director for Hemispherx Biopharma, Inc. Hemispherx Biopharma, based in Philadelphia, is a biopharmaceutical company engaged in the manufacture and clinical development of new drug entities for treatment of viral and immune-based disorders. Hemispherx’s flagship products include Alferon N Injection® and the experimental immunotherapeutics/antivirals Ampligen®. More information One Penn Center 1617 JFK Blvd., 6th Floor Philadelphia, PA 19103 Phone: (215) 988-0080 Fax: (215) 988-1739 E-mail: info@hemispherx.net retrospectively monitor blood from patients enrolled in the key pivotal study, AMP-516. The primary purpose of this study protocol is to identify target subsets of patients who are most likely to benefit from active treatment with Ampligen®. This study specifically is monitoring patients for evidence of xenotropic murine leukemia virusrelated virus (XMRV) which has recently been implicated as having a strong association with CFS. One of the key research organizations, The Whittmore Peterson Institute, is performing the analysis for this on-going study. We anticipate that this data will be available for analysis by late summer. The information gained from this study and the recommendations made by the FDA in the complete response letter along with their review of the new subgroup analysis, when completed, will guide the design of the next clinical study using Ampligen® to treat CFS. This article will review the findings of a number of clinical studies which explore the use Ampligen® in Page 32/56

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