Down-regulation of MYCN protein by CX-5461 leads to neuroblastoma tumor growth suppression

Preclinical study demonstrates CX-5461, an RNA polymerase I inhibitor, suppresses neuroblastoma tumor growth by down-regulating MYCN/MYC proteins. Local delivery via silk film platform showed significant tumor growth delay in MYCN-amplified orthotopic mouse models, suggesting potential therapeutic strategy for high-risk neuroblastoma.