1.Telomerase Inhibitor Imetelstat in Patients with Essential Thrombocythemia.
Baerlocher GM1, Oppliger Leibundgut E, Ottmann OG, Spitzer G, Odenike O, McDevitt MA, Röth A, Daskalakis M, Burington B, Stuart M, Snyder DS. N Engl J Med. 2015 Sep 3;373(10):920-8. doi: 10.1056/NEJMoa1503479.
BACKGROUND: Imetelstat, a 13-mer oligonucleotide that is covalently modified with lipid extensions, competitively inhibits telomerase enzymatic activity. It has been shown to inhibit megakaryocytic proliferation in vitro in cells obtained from patients with essential thrombocythemia. In this phase 2 study, we investigated whether imetelstat could elicit hematologic and molecular responses in patients with essential thrombocythemia who had not had a response to or who had had unacceptable side effects from prior therapies.