Breakthrough in treatment of devastating neuroblastoma cancer

A NEW class of drugs is showing signs of overcoming some of the most devastating forms of childhood cancer following groundbreaking Australian results.

Using a combination of two new drugs, Australian researchers have been able to cure highrisk neuroblastoma in animals – a disease that claims the lives of more than half the children diagnosed with it within five years.

Initial Australian results have been so promising that trials of one of the drugs, called CBL137, will see it given to children suffering neuroblastoma at more than 100 hospitals around the world from September.

But further findings from the Children’s Cancer Institute released this week show its impact can be multiplied when combined with another emerging drug called panobinostat, fuelling hopes of expanding the international trial. Senior researcher Professor Michelle Haber of the Children’s Cancer Institute said the two drugs both worked to disrupt the relationship between the DNA and proteins of a tumour cell to deliver “a supercharged hit that it can’t survive”.

“We found when we put these together we got a number of very exciting outcomes, all of which were positive in terms of killing the tumour cells,” Prof Haber said. “Together we were able to get a complete tumour regression.”

About 40 Australian children are diagnosed with neuroblastoma each year. For more than 40 years chemotherapy drugs have been used to destroy the DNA of neuroblastoma and other cancer cells to kill off the diseases.

But the toxic drugs also destroy other vital cells of the body causing severe consequences, while also allowing the disease a chance to evolve and often come back.

Unlike chemotherapy, the experimental CBL137 and panobinostat therapies instead target chromatin, a combination of DNA and proteins that control which genes the cancer cell switches on and off.

By disrupting the relationship between the DNA and the proteins the therapies prevent cancer cells flicking the switch that allows them to grow and multiply.

Results published in the Clinical Cancer Research journal found the combined therapies not only stopped neuroblastoma in its tracks in mice, but avoided all the wider DNA damage caused by ‘chemo’.

The research is expected to attract international attention because it may also open up new ways to target other types of childhood cancers.