Laboratory studies in the US have confirmed the promise of an experimental drug against a particularly aggressive form of neuroblastoma, the most common non-blood cancer in children.

In work published in the journal Science Translational Medicine, William Weiss and colleagues at the University of California in San Francisco found that a drug called NVP-BEZ235 could prolong survival in mice with the disease.

Neuroblastoma is caused when nerve cells called neuroblasts develop into cancer cells rather than working nerve cells. These cells then form tumours in the brain or peripheral nerves.

Since 1971, survival rates from neuroblastoma have risen from 17 to 64 per cent. But despite this progress, the aggressive form of the disease is still very hard to treat successfully.

Decades of research have linked aggressive, or 'high-risk', neuroblastoma to an overactive gene called MYCN. Researchers have been trying to exploit this fact to develop new treatments.

Previous research had suggested that NVP-BEZ235, a new experimental drug, might be suitable to target cells that are driven by overactive MYCN.

In a series of detailed experiments, the researchers showed that NVP-BEZ235 caused the breakdown of MYCN in the neuroblastoma cells of mice, which in turn blocked new blood vessels forming in the tumours.

By halting the blood supply that the cancer needs to grow, the mice survived longer.

Clinical trials will now be needed to see if NVP-BEZ235 is as effective in children with aggressive neuroblastoma.

UK experts welcomed the research. Professor Andy Pearson, Cancer Research UK Professor of Paediatric Oncology at The Institute of Cancer Research and The Royal Marsden NHS Foundation Trust, said: "We urgently need better ways to help those children with aggressive neuroblastoma, as this type is very difficult to treat and is a major cause of childhood cancer deaths.

"For almost 20 years, scientists have been able to tell whether a child's tumour is high-risk by looking for faults in a gene called MYCN in the tumour cells. But they haven't been able to work out how to use this knowledge to develop new targeted treatments."

However, he cautioned that the work was still at a preliminary stage.

"This new work is at an early stage, and not yet appropriate for patients. But it's among a growing body of exciting research showing that we can interfere with the effects of the MYCN gene to treat neuroblastoma," he added.

"Work like this shows how we're moving away from conventional chemotherapy towards more targeted therapy in neuroblastoma - as we are in other types of cancer. Cancer Research UK is funding several key laboratory projects and early clinical trials in neuroblastoma and other childhood cancers, which are looking into similar targeted treatments."

Source: CRUK