Imetelstat is effective in Acute Myeloid Leukemia (AML) patient xenografts.

Imetelstat is a covalently lipidated 13-mer thiophosphoramidate oligonucleotide that competitively inhibits telomerase activity. Here, we show that imetelstat effectively diminishes AML burden and prolongs survival in a comprehensive, randomized Phase II-like preclinical trial in AML patient-derived xenografts (PDX). Overall design: RNA profiles of splenic hCD45+ cells from NRGS mice transplanted with AML patient samples (8 individual models), generated after treatment with vehicle or imetelstat, in duplicate.