Neuroblastoma, a common pediatric solid tumor, has a poor prognosis for high-risk cases. This study investigated indisulam, a drug showing sensitivity in neuroblastoma cell lines. Indisulam targets RNA splicing factor RBM39, causing its degradation and leading to splicing errors, growth inhibition, and metabolic disruption. In vivo studies using xenograft and transgenic models demonstrated complete tumor regression without relapse. The dual targeting of metabolism and RNA splicing by indisulam offers a promising therapeutic approach for high-risk neuroblastoma.
Publisher
Nature Communications
Published On
Mar 16, 2022
Authors
Anke Nijhuis, Arti Sikka, Orli Yogez, Lili Herendi, Cristina Balcells, Yurui Ma, Evon Poon, Clare Eckold, Gabriel N. Valbuena, Yuewei Xu, Yusong Liu, Barbara Martins da Costa, Michael Gruet, Chiharu Wickremesinghe, Adrian Benito, Holger Kramer, Alex Montoya, David Carling, Elizabeth J. Want, Yann Jamin, Louis Chesler, Hector C. Keun
Tags
neuroblastoma
indisulam
RNA splicing
tumor regression
pediatric cancer
metabolic disruption
high-risk
Related Publications
Explore these studies to deepen your understanding of the subject.
