%0 Journal Article %A Mitchell, William M %D 2016 %T Efficacy of rintatolimod in the treatment of chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME) %U https://tandf.figshare.com/articles/journal_contribution/Efficacy_of_rintatolimod_in_the_treatment_of_chronic_fatigue_syndrome_myalgic_encephalomyelitis_CFS_ME_/3438866 %R 10.6084/m9.figshare.3438866.v1 %2 https://tandf.figshare.com/ndownloader/files/5398442 %K Rintatolimod %K chronic fatigue/myalgic encephalomyelitis %K TLR3 agonist %K clinical trials %K Ampligen %K dsRNA %K clinical efficacy %K clinical safety %K primate/non-primate disassociation of toxicity %X

Chronic fatigue syndrome/ Myalgic encephalomyelitis (CFS/ME) is a poorly understood seriously debilitating disorder in which disabling fatigue is an universal symptom in combination with a variety of variable symptoms. The only drug in advanced clinical development is rintatolimod, a mismatched double stranded polymer of RNA (dsRNA). Rintatolimod is a restricted Toll-Like Receptor 3 (TLR3) agonist lacking activation of other primary cellular inducers of innate immunity (e.g.- cytosolic helicases). Rintatolimod also activates interferon induced proteins that require dsRNA for activity (e.g.- 2ʹ-5ʹ adenylate synthetase, protein kinase R). Rintatolimod has achieved statistically significant improvements in primary endpoints in Phase II and Phase III double-blind, randomized, placebo-controlled clinical trials with a generally well tolerated safety profile and supported by open-label trials in the United States and Europe. The chemistry, mechanism of action, clinical trial data, and current regulatory status of rintatolimod for CFS/ME including current evidence for etiology of the syndrome are reviewed.

%I Taylor & Francis