Neuroblastoma is a heterogenous and frequently lethal childhood cancer. Using tumor organoids from MYCN-amplified high-risk neuroblastomas, Hansson and colleagues performed a high-throughput screen using approved or emerging cancer drugs. The authors identified several novel compounds specifically affecting neuroblastoma organoids. One of these was ARRY-520, an inhibitor of kinesin spindle protein (KSP). Importantly, Hansson and colleagues found that high expression of KIF11, the gene encoding KSP, was correlated with poor outcome in neuroblastoma patients but not in 21 other human cancers. Inhibition of KSP in patient-derived xenografts (PDX) from neuroblastoma caused numerous abnormalities during cell division, leading to tumor cell apoptosis. The authors further showed that inhibition of KSP resulted in decreased tumor burden and enhanced survival mice with MYCN-amplified neuroblastoma PDX tumors in vivo.

Expert Commentary: These data suggest that inhibition of KSP could be an attractive strategy for treating patients with high-risk neuroblastoma.

Hansson K,...

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