Tumour-targeted drugs tested in children with high grade brain tumours

Testing three new drugs in children with DIPG brain tumours, with drugs selected according to tumour subtype.

Dr Darren Hargrave and his team at Great Ormond Street Hospital hope to identify new drugs which will improve survival and quality of life for children affected by DIPG brain tumours. All patients participating in this clinical trial could benefit due to it's new, flexible design in which three drugs are tested at once.

The trial is for patients with a Diffuse Intrinsic Pontine Glioma, or DIPG, which is the most aggressive brain tumour in children. Radiotherapy is currently the only proven treatment for this tumour type, and is essentially palliative, meaning there is an urgent need for new, effective treatments.

In this trial, co-funded with Cancer Research UK, Dr Hargrave's team will test one of three new drugs in 150 children with DIPG over five years. In each case they will assess whether the drug is safe, and effective, when combined with radiotherapy.

Crucially, the drug used to treat each patient will be selected according to the molecular subtype of their particular tumour, which will be determined in advance via biopsy.

The trial's flexible 'adaptive' design means that if an interim analysis reveals that one or more of the drugs does not appear to be working it will be dropped in favour of an alternative.

Results for children on the trial will be compared with those achieved previously using standard treatment alone to determine whether any of the drugs prolong survival.

The three drugs erlotinib, desatinib and everolimus all target cancer cells in different ways and are currently used successfully in the treatment of other cancers.

Project information

Formal title:Biological Medicine for Diffuse Intrinsic Pontine Glioma (DIPG) Eradication (BIOMEDE)

Key Researcher: Dr Darren Hargrave, Great Ormond Street Hospital
for children

Tumour type: Diffuse Instrinsic Pontine Glioma (DIPG)

Research type: Clinical trial

Timing: Granted in April 2015 for five years