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Targeted Neuroblastoma Therapy One Step Closer

By Drug Discovery Trends Editor | August 27, 2014

neuroblastomaAustralian researchers have uncovered new targets for therapy in the childhood cancer neuroblastoma. Tao Liu, a researcher from the Histone Modification Group and Children’s Cancer Institute Australia for Medical Research, said the researchers have been studying neuroblastoma in the hope of developing novel treatments.
“Neuroblastoma is the most common solid tumor in early childhood and it accounts for 15% of all childhood cancer death,” Liu said. “In Australia, cancer is the most common cause of death from disease for children, and neuroblastoma is one of the most devastating types.”
Neuroblastoma affects the peripheral nervous system in children, and, in most cases, presents in the adrenal glands, found above the kidneys.
Gene expression key to tumor severity
Children who have neuroblastoma have higher expression of a specific gene, which intensifies disease progression. Now, researchers have identified the mechanism responsible for expressing this gene.
“Normally humans only have two copies of N-Myc gene but in children who have neuroblastoma they have 100-150 copies,” Liu said.
The research found a novel long noncoding RNA was responsible for increasing expression of the gene and promoting the progression of tumors.
“We have confirmed the novel long noncoding RNA amplifies the expression of N-Myc oncogene,” Liu said. “The novel long noncoding RNA is critical for the expression of N-Myc oncogene. It’s critical for tumor progression… [and] it has other pathways it can affect that are independent of N-Myc.”
Liu added that many different types of cancers have become treatable in the last 30 years, but neuroblastoma still has a high mortality rate.
Children who suffer from “neuroblastoma with amplification of the N-Myc oncogene are much more likely to die of the disease,” Liu said.
Targeting the long noncoding RNA for therapy
Principal Investigator for the Harry Perkins Institute of Medical Research and the University of Western Australia, Archa Fox, said there are two new findings: the novel long noncoding RNA and the NonO protein, which along with N-Myc, contributes to severity of the cancer.
“NonO is an oncoprotein in this cancer, it’s a bad guy and we are using our knowledge and tools to work on targeting it therapeutically,” Fox said.
High levels of novel long noncoding RNA and NonO expression in children who suffer from neuroblastoma, results in poor patient outcome.
Fox also said they are both new targets that the researchers can potentially create drugs to inhibit. “If they are inhibited it will slow down and hopefully kill the cancer cells, this should make the world of difference for these children with this type of neuroblastoma,” she said.
Date: August 27, 2014
Source: Science Network WA

Filed Under: Drug Discovery

 

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