Anti-Nucleolin Aptamer iSN04 Inhibits Alveolar Rhabdomyosarcoma Cell Growth by Inducing Myogenic Differentiation

iSN04 treatment induces differentiation in NRS-1 cells and severely impairs RH30 alveolar rhabdomyosarcoma tumor-sphere formation, with cooperative growth suppression when combined with low-dose vincristine.

• Why it matters: Standard chemotherapy like vincristine has only marginally improved survival for high-risk rhabdomyosarcoma, creating a need for novel agents that target tumor growth and differentiation.
• What they did: The study tested the anti-nucleolin aptamer iSN04 in two alveolar RMS cell lines, measuring gene expression changes in MyoD, myogenin, ID1, ID3, and CASZ1, and assessing 3D tumor-sphere growth.
• The result: iSN04 drove NRS-1 differentiation into contracting myotube-like cells and severely impaired RH30 tumor-sphere formation, and co-treatment with iSN04 plus low-dose VCR cooperatively suppressed RH30 proliferation versus single agents.