A near-hopeless childhood cancer succumbs to drug duo


Cells from a diffuse intrinsic pontine glioma, a fatal childhood cancer. Lab tests have identified a drug combination that effectively attacks the class of tumours that includes this glioma. Credit: Shawn Gillespie, Monje Lab


Mass screening turns up a therapy that holds promise for treating a highly aggressive class of tumours.

A combination of drugs has shown promise in laboratory tests against an inevitably fatal nervous-system cancer that mostly strikes children.

Diffuse midline gliomas are tumours of the central brain and spinal cord. The only available treatment is radiation, and the median survival for one type of this cancer, diffuse intrinsic pontine glioma, is 9–11 months. Previous work has suggested that the drug panobinostat kills these tumour cells, but they ultimately become resistant to the treatment.

Michelle Monje at Stanford University in California and her colleagues looked for drugs that could be used alone or in combination to kill diffuse midline glioma cells. The team tested 2,706 single compounds and 9,195 drug combinations in cell cultures grown from patients’ tumours.

A combination of two drugs, panobinostat and marizomib, increased survival in mice bearing tumours grown from the samples. This drug combination altered when genes in the cancer cells turn on and off, and interfered with cellular metabolism.

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