Transcriptionally distinct malignant neuroblastoma populations show selective response to adavosertib treatment.

ABSTRACT: Neuroblastoma is an aggressive childhood cancer that arises from the sympathetic nervous system. Despite advances in treatment, high-risk neuroblastoma remains difficult to manage due to its heterogeneous nature and frequent development of drug resistance. Drug repurposing guided by single-cell analysis presents a promising strategy for identifying new therapeutic options. Here, we aim to characterize high-risk neuroblastoma subpopulations and identify effective repurposed drugs for targeted treatment. We performed single-cell transcriptomic analysis of neuroblastoma samples, integrating bulk RNA-seq data deconvolution with clinical outcomes to define distinct malignant cell states. Using a systematic drug repurposing pipeline, we identified and validated potential therapeutic agents targe

SUBMITTER: Hsieh CH 

PROVIDER: S-EPMC12047484 | biostudies-literature | 2025 Apr

REPOSITORIES: biostudies-literature