Promising developments in the treatment of rare childhood cancer

The combination of two drugs yields promising results in the treatment of neuroblastoma, a rare but serious form of cancer that mainly affects babies and young children. This was discovered by researchers from the Pediatric Precision Oncology Lab (PPOL) at Ghent University, in cooperation with the Dutch Princes Máxima Children's Cancer Centre and the American Memorial Sloan Kettering Cancer Center. The key to the new treatment lies with an existing drug: Triapine.

In neuroblastoma, a malignant cancer develops on a nerve node, for example in the abdomen at the level of the adrenal gland. The cancer cells spread through the body via blood and lymph vessels. Triapine inhibits the production of DNA building blocks, making the tumour cells more sensitive to another drug, Prexasertib. Neuroblastoma cells react very strongly to the combination of Triapine and Prexasertib, even at low concentrations and both in cell culture and in animal experiments.

A European collaboration is therefore underway to evaluate the new treatment strategy in children with neuroblastoma. Other types of childhood cancer may also be better treated with the drug in the future.

The UGent researchers have also discovered a biological mechanism that is specific to patients with high-risk neuroblastoma, namely the increased production of DNA that makes the cancer cells grow faster. Existing medication is insufficient for a good recovery.

The research was realised with financial support from Kom op tegen Kanker, the Olivia Fonds, the Kinderkankerfonds and the Villa Joep Fonds.




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