Neuroblastoma UK, a national research charity, has welcomed the news that a new treatment for children with neuroblastoma will be advanced into clinical trials.
The drug, which has already passed safety trials in adults, indirectly targets N-Myc, a gene that occurs in aggressive forms of neuroblastoma.
The treatment works by blocking N-Myc activity by switching off the production of the gene, with these promising results leading to the launch of a clinical trial in children with high-risk neuroblastoma by the end of 2020.
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.
The study of the treatment – fadraciclib – was led by scientists at the Institute of Cancer Research and were published in The Journal of Clinical Investigation earlier this week.
“We welcome the news that a potential new treatment will be advanced to clinical trial and reach more young cancer patients,” said Tony Heddon, chair of Neuroblastoma UK.
“Less than 50% of children with high-risk neuroblastoma will survive for five years or more after their diagnosis. And for those children who do survive, the drugs used to save them may cause long-lasting damage.
“There is a real need to provide less toxic and more effective treatment for our children. This important development offers real hope to families affected by this life-threatening cancer,” he added.