An experimental drug, currently in clinical trials for adult cancer, has the potential to halt tumour progression of a cancer of the nervous system in children, according to a team of Australian scientists.

The new study from Children’s Cancer Institute also found that the drug, known as CBL0137, could block the very start of this embryonal cancer called neuroblastoma, which can pave the way to possible prevention strategies in the future. CBL0137 was developed by a team from Roswell Park Cancer Institute in the U.S., long-term collaborators with Children’s Cancer Institute.

When used in combination with traditional DNA-damaging chemotherapy agents, the drug was much more effective than either drug alone, the researchers say in the study published in Science Translational Medicine. This is because CBL0137 prevents the cancer cells from repairing DNA damage induced by chemotherapy, which ensures cell death.

“Our laboratory tests tell us that CBL0137 is likely to be very effective against the most aggressive neuroblastomas, and indeed the most aggressive forms of other childhood cancers, and that is very exciting,” said Professor Michelle Haber, one of the study’s authors and the executive director of Children’s Cancer Institute.

In contrast to many other chemotherapeutic agents, the drug also does not damage DNA, according to Haber. She added that DNA damage is responsible for the many unpleasant and serious side-effects that frequently affect children after they are cured of their cancer.

For CBL0137 to be effective at preventing neuroblastoma, it would have to be given to all children at birth. “If prevention is to be more than a pipedream, the medicine would have to be completely safe. You would give it once or twice to a newborn, as you give vitamin K, as a way of killing off excess cells that should have died prior to birth,” said Professor Glenn Marshall, director of the Kids Cancer Centre at Sydney Children’s Hospital, Randwick and head of Translational Research at Children’s Cancer Institute.

CBL0137 is currently in Phase 1 clinical trials for adults, which means that safe dosage levels are being tested, says Haber. Once the adult trials are completed, a Phase 1 trial for children with refractory or relapsed neuroblastoma, and also other aggressive childhood cancers, will open in Australia and the U.S.

The drug’s clinical trial in children will be conducted at leading children’s cancer centres at Sydney Children’s Hospital, Randwick, through the US-based Children’s Oncology Group, the largest children’s cancer study group in the world. This is the first time that a COG trial of this sort would be made available to Australian children.

Neuroblastoma is a form of cancer that is made up of cells that are found in nerve tissues of the body, according to Cancer Australia. It occurs most commonly in infants and children under five years of age, and rarely in children over 10 years of age.

Considered the most common solid tumour of early childhood, neuroblastoma is generally diagnosed when the disease is advanced. Around half of all children with neuroblastoma have aggressive tumours, and fewer than half of these patients survive even after intensive treatment.

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