Medicine and Health

Indisulam targets RNA splicing and metabolism to serve as a therapeutic strategy for high-risk neuroblastoma

A. Nijhuis, A. Sikka, et al.

Discover how researchers Anke Nijhuis and colleagues have unleashed the potential of indisulam, a drug targeting RNA splicing factor RBM39, to combat high-risk neuroblastoma. Their groundbreaking study showcases complete tumor regression in xenograft models, reshaping our approach to pediatric cancer treatment.

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Abstract

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

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