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2016 ; 9
(6
): 755-70
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Efficacy of rintatolimod in the treatment of chronic fatigue syndrome/myalgic
encephalomyelitis (CFS/ME)
#MMPMID27045557
Mitchell WM
Expert Rev Clin Pharmacol
2016[Jun]; 9
(6
): 755-70
PMID27045557
show ga
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.- 2'-5' 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.