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New drug shows potential for treating childhood neuroblastoma

Cancer Research UK-funded scientists have found a new drug that is likely to be effective against the aggressive childhood cancer neuroblastoma.

Neuroblastoma is a type of nerve tumour which is difficult to treat and often reoccurs after intensive treatment. Existing therapies for this type of cancer are particularly intense and have a profound impact on young patients and often cause long-term side effects.

Scientists at The Institute of Cancer Research, London, have discovered a new drug – fadraciclib – which is effective at blocking N-Myc activity.

Changes in the N-Myc gene occur in aggressive forms of neuroblastoma, making it a good target for treatment in this rare cancer type.

Fadraciclib has already passed safety trials in adults, and was jointly discovered by scientists at the ICR in collaboration with Cyclacel. The drug works by switching off the production of N-Myc.

In mice, the drug slowed down and stabilised tumour growth, and the mice who received it also survived for longer after treatment than untreated mice. When combined with chemotherapy in mice, the drugs shrank the tumours to the point of virtually eradicating them.

“I’m incredibly proud of the work our researchers have done to provide this hope for children with neuroblastoma,” said Michelle Mitchell, chief executive at Cancer Research UK.

“Children with aggressive cancer often go through gruelling treatments that, even when successful, may cause long-term side-effects. They still have their whole lives ahead of them, so it’s paramount to develop alternatives that won’t affect their quality of life in the long run,” she added.