The MYCN 5′ UTR as a therapeutic target in neuroblastoma
MYCN amplification is a key driver of high-risk neuroblastoma. Volegova et al. capitalize on the dependence of MYCN-amplified neuroblastoma cells on increased protein synthesis by using amidino-rocaglates to inhibit translation initiation factor eIF4A1, resulting in direct targeting of the MYCN mRNA and selective cytotoxicity in animal models.