Child brain tumour drug launched

A drug that can shrink brain tumours by up to 50 per cent in children with a rare disease has been launched in the UK.

NHS doctors will be able to apply for funding from health trusts for Votubia (everolimus) for children with growing non-malignant brain tumours associated with a condition called tuberous sclerosis complex.

The once-a-day pill offers an alternative to surgery and could provide a lifeline for up to 1,600 youngsters in the UK with the tumours.

The drug, which has been granted orphan drug status for rare diseases, has been shown to shrink subependymal giant cell astrocytoma tumours associated with TSC.

TSC is a genetic disorder which leads to non-malignant tumours forming in organs, most commonly the brain and kidneys.

Brain tumours occur in up to 20 per cent of patients with TSC, causing physical and mental disability.

Chris Kingswood, head of research at the Tuberous Sclerosis Association, said: “For a long time there has been a desperate need for a useful alternative licensed treatment to invasive brain surgery.

“The way it works in the signalling pathway can be simply likened to blocking a receiver so it cannot process signals from the aerial on an analogue radio, so that the signals can no longer transmit to drive in this case tumour cell growth.”

Finbar O’Callaghan, consultant in paediatric neurology at the University of Bristol, said: “SEGA is a serious complication in TSC, accounting for much of the increased mortality seen in this condition.

“Until now, brain surgery has been the only option for treatment and the availability of a pharmacological therapy is a major milestone and provides a treatment option in those cases where surgery is difficult or not possible.”