title: Further developments with antisense treatment for myasthenia gravis.
abstract: We present further developments in the study of the antisense oligonucleotide EN101. Ongoing in vitro and in vivo studies demonstrate that EN101 is a TLR9-specific ligand that can suppress pro-inflammatory functions and shift nuclear factor kappa B (NF-κB) from the pro-inflammatory canonical pathway to the anti-inflammatory alternative pathway, which results in decreases acetylcholinesterase (AChE) activity. Preliminary results of a double-blinded phase II cross-over study compared 10, 20, and 40 mg EN101 administered to patients with myasthenia gravis. Patients were randomly assigned to one of three treatment groups in weeks 1, 3, and 5 and received their pretreatment dose of pyridostigmine in weeks 2 and 4. Thus far, all doses show a decrease in QMG scores, with a greater response to higher doses.
authors.affiliation: Greater Manchester Neuroscience Centre, Manchester UK. jon.sussman@manchester.ac.uk
authors.forename: Jon
authors.initials: J
authors.lastname: Sussman
authors.forename: Zohar
authors.initials: Z
authors.lastname: Argov
authors.forename: Yitzhak
authors.initials: Y
authors.lastname: Wirguin
authors.forename: Slobodan
authors.initials: S
authors.lastname: Apolski
authors.forename: Vedrana
authors.initials: V
authors.lastname: Milic-Rasic
authors.forename: Hermona
authors.initials: H
authors.lastname: Soreq
mesh_terms: D000110:Acetylcholinesterase / Q000097:blood; D000818:Animals; D018592:Cross-Over Studies; D004311:Double-Blind Method; D006801:Humans; D009157:Myasthenia Gravis / Q000188:drug therapy*; D009838:Oligodeoxyribonucleotides / Q000627:therapeutic use*; D016376:Oligonucleotides, Antisense / Q000627:therapeutic use*; D051381:Rats
chemical_list: C477243:EN101; D009838:Oligodeoxyribonucleotides; D016376:Oligonucleotides, Antisense; D000110:Acetylcholinesterase