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Extra copies of the MYCN oncogene, found in about 20% of neuroblastomas, mark the most aggressive disease and define high risk at any age.
MYCN amplification means extra copies of the MYCN oncogene, detected by FISH when a cell carries more than 10 copies, and it marks the most aggressive form of neuroblastoma, defining high-risk disease at any age. It is found in a minority of neuroblastomas and is associated with rapid progression, 1p deletion and poor outcome. MYCN itself is not directly druggable, so the approaches are indirect: BET and Aurora A inhibitors, ODC1 and polyamine inhibition with eflornithine (DFMO), which acts on the MYCN pathway, and targeting ALK co-mutations with lorlatinib in ANBL1531. Readers meet the term in the neuroblastoma record, the eflornithine drug record, the MYC pathway record, the profile of Susan L. Cohn, and the bottleneck on rare and paediatric cancers without markets.
Showing the molecule this term concerns: Eflornithine (DFMO).
The glossary entry explains the word; the readout page carries the scoring rule, the thresholds approvals use, the companion diagnostics and the tests.
Shares Susan L. Cohn, Segmental chromosomal aberrations and ploidy (neuroblastoma), Low-risk neuroblastoma (INRG very low and low risk, including stage MS), Intermediate-risk neuroblastoma.
Shares Eflornithine (DFMO), High-risk neuroblastoma, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
Shares Low-risk neuroblastoma (INRG very low and low risk, including stage MS), Intermediate-risk neuroblastoma, High-risk neuroblastoma.
Shares Low-risk neuroblastoma (INRG very low and low risk, including stage MS), Intermediate-risk neuroblastoma, High-risk neuroblastoma, Neuroblastoma (paediatric).
Shares Eflornithine (DFMO), High-risk neuroblastoma, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
Shares Low-risk neuroblastoma (INRG very low and low risk, including stage MS), Intermediate-risk neuroblastoma, High-risk neuroblastoma, Neuroblastoma (paediatric).
Shares Ependymoma molecular groups (PF-A, PF-B, ZFTA, YAP1, MYCN), Medulloblastoma molecular groups (WNT, SHH, group 3, group 4).
Shares High-risk neuroblastoma, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.