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In vitro and cell-based studies suggest combining an inhibitor of nonsense-mediated RNA decay and a promoter of premature stop-codon read-through could help treat cancer. Virtual library screening and in vitro testing identified several nonsense-mediated RNA decay inhibitors with nanomolar potency. In human cancer cell lines harboring premature stop-codon mutations in p53, one inhibitor increased mutant p53 mRNA levels compared with vehicle. In the cancer cell lines, the inhibitor and a research compound that promotes premature stop-codon read-through synergistically increased functional p53 levels and cell death compared with either agent alone. Next steps include optimizing the nonsense-mediated RNA decay inhibitors.
PTC Therapeutics Inc.'s Translarna ataluren (formerly PTC124), a small molecule that facilitates complete translation of proteins containing nonsense mutations, is under EMA review to treat muscular dystrophy. The compound also is in Phase III testing to treat cystic fibrosis (CF) and Phase II trials to treat methylmalonic acidemia.

SciBX 7(17); doi:10.1038/scibx.2014.485
Published online May 1, 2014

Patented by New York University; unlicensed; available for partnering

Martin, L. et al. Cancer Res.; published online March 24, 2014;
Contact: Lawrence B. Gardner, New York University School of Medicine, New York, N.Y.