Research ArticleCancer

Targeted inhibition of histone H3K27 demethylation is effective in high-risk neuroblastoma

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Science Translational Medicine  16 May 2018:
Vol. 10, Issue 441, eaao4680
DOI: 10.1126/scitranslmed.aao4680

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A different way to differentiate neuroblastoma

Neuroblastoma is one of the more common pediatric solid tumors, and it can be difficult to treat. These tumors are characterized by the failure of neural crest precursor cells to differentiate and can sometimes be treated with compounds that induce differentiation. Unfortunately, these do not always work, and thus, Lochmann et al. performed a high-throughput drug screen in search of additional differentiating agents, particularly ones that work by altering tumor epigenetics. Through such screening, the authors identified a compound called GSK-J4, which inhibited histone demethylation, induced tumor cell differentiation, and blocked tumor growth in multiple models of neuroblastoma. In addition, GSK-J4 cooperated with two approved drugs, suggesting that it could be a valuable part of treatment for this lethal disease.