Targeted Disruption of MDM2 Dimerisation as a Novel Approach to Treating High-Risk Neuroblastoma

Accounting for greater than 20% of neonatal cancers, neuroblastoma (NB) represents the most common extracranial paediatric malignancy [1].

An embryonal tumour, NB arises from the sympathetic nervous system and is responsible for 15% of paediatric cancer mortality.

Despite significant advances in NB diagnosis and treatment, high-risk NB (i.e., metastatic disease over 1-year of age or MYCN-amplified disease) has a 5-year overall survival of less than 50%.

NB is a highly heterogenous disease, relying significantly upon available next-generation precision medicine tools to characterise genomic / transcriptomic / proteomic profiles, and better stratify patients into specific sub-groups which are then associated with the appropriate treatment strategies.

Despite this, high-risk NB (accounting for 50% of NB cases) is highly drug resistant and patients persistently relapse, re-enforcing an urgent need for novel therapeutic interventions aimed at improving treatment response and overall survival.