New gene found for a deadly childhood cancer offers possibility of targeted drug therapy

Neuroblastoma accounts for 15% of total childhood cancer deaths. The survival rate of high-risk neuroblastoma patients is 50%.

For the first time, Australian researchers from Children’s Cancer Institute have discovered that a gene called JMJD6 plays an important role in the most aggressive form of the disease. The research is published today in the journal, Nature Communications.

The discovery opens up a new way to treat neuroblastoma, using drugs that target this gene.

It has long been known that a gain of the chromosomal region 17q is associated with the form of neuroblastoma with the poorer prognosis, however the particular genes within that chromosomal region that are important in neuroblastoma—and therefore are potential drug targets—have until now eluded researchers.

Associate Professor Tao Liu and his research team from Children’s Cancer Institute found a candidate gene, called JMJD6, from a tumour data base of 209 patients, finding the gene active in more than one in four patients with the aggressive form of neuroblastoma.

They turned off the gene in neuroblastoma cell lines and animal models of the disease and found that this led to a:

  • Reduction in the growth of neuroblastoma cells
  • Reduction in tumour progression
  • Increased survival

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