Integrative Bioinformatics Analysis of Dyseregulated Pathways in Neuroblastoma to Identify Repurposable FDA-Approved Drug Candidates

Author(s)	Mr. Korounganba Rajkumar
Country	India
Abstract	High-risk neuroblastoma (NB) remains one of the most difficult pediatric cancers to treat, particularly in patients with MYCN amplification, where survival outcomes have remained relatively stagnant despite aggressive multimodal therapy. In this project, I focused on understanding how MYCN amplification alters tumor biology at the transcriptomic level and whether these changes reveal actionable therapeutic vulnerabilities. Using RNA-seq data from the TARGET-NBL cohort, I performed a differential expression analysis comparing MYCN-amplified and non-amplified high-risk tumors. I identified 1,842 significantly dysregulated genes, including a consistent 3.4-fold overexpression of ODC1 in MYCN-amplified samples. I then integrated this disease signature with LINCS L1000 perturbation profiles to identify FDA-approved drugs capable of reversing the observed expression pattern. This analysis highlighted Eflornithine (DFMO) as a strong candidate targeting polyamine metabolism. To better understand why such computational findings rarely translate into clinical use, I also conducted a structured survey of pediatric oncology professionals to assess real-world translational barriers.
Published In	Volume 8, Issue 1, January-February 2026
Published On	2026-02-05
DOI	https://doi.org/10.36948/ijfmr.2026.v08i01.68090

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