A First-in-Human Phase 1 Study of a Tumor-Directed RNA-Interference Drug against HIF2α in Patients with Advanced Clear Cell Renal Cell Carcinoma.

James Brugarolas Gregory Obara Kathryn E Beckermann Brian I Rini Elaine T Lam James Hamilton Thomas Schluep Min Yi So C Wong Zhongping Lily Mao Erick Gamelin Nizar M Tannir

Published in: Clinical cancer research : an official journal of the American Association for Cancer Research (2024)

ARO-HIF2 downregulated HIF2α in advanced ccRCC-inhibiting tumor growth in a subset of subjects. Further development was hampered by off-target neurotoxicity and low response rate. This study provides proof of concept that siRNA can target tumors in a specific manner.

Keyphrases

endothelial cells
open label
randomized controlled trial
emergency department
pluripotent stem cells