Study discovers targeted therapy to help adolescents with deadly nerve cancer

A targeted therapy has been discovered to treat adolescent patients who suffer from a deadly paediatric nerve cancer called neuroblastoma, a recent study has stated.


Neuroblastoma is one of the most common and aggressive paediatric nervous system tumours and generally has a poor prognosis, particularly when it advances in older children.

Treatment success for the disease varies, but is exponentially less in adolescent patients, particularly because the disease lacks effective targeted therapies.

The Mount Sinai researchers found that neuroblastoma in older children and adolescents harbouring deletions within a gene called ATRX may be responsive to a targeted therapy called tazemetostat.

Tazemetostat disables an enzyme called EZH2 that inhibits genes that promote normal neuron development, in turn killing neuroblastoma cells. Neuroblastoma arises in immature nerve cells of the adrenal glands and portions of the spine during the development of the sympathetic nervous system, which controls the body's "flight or fight" response to stress.

EZH2 inhibitors are already being tested in phase I and phase II clinical trials for other cancers, including lymphomas, sarcomas, and other solid tumours, with some favourable results.

"We hypothesised that mutant ATRX proteins contribute to aggressive neuroblastoma," said a Professor of Oncological Sciences and senior corresponding author.

"In this study, we aimed to decipher the underlying biology of these altered proteins in neuroblastoma, a tumor for which effective therapeutic strategies remain obscure, and to exploit identified dependencies," added the Prof.

Mount Sinai scientists continue to expand this research into the role of the mutant ATRX protein in the laboratory and hope to eventually open a clinical trial with collaborating institutions.

Based on this research, they believe that EZH2 inhibitors could also be effective in other ATRX mutant cancers, such as pediatric glioblastoma, multiforme and osteosarcoma.

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