Brain cancer experts crowdsource a cure for deadly glioblastoma

A new approach to brain cancer research

Clinicians and researchers from cancer centres in Australia and around the world have just met at an event in Washington to start crowdsourcing a way to design an ambitious clinical platform aimed at rapidly identifying a cure for the world's deadliest and most common brain cancer, glioblastoma.

"This will be a Silicon Valley approach to medical research," says medical oncologist and CTC senior research fellow Dr Mustafa Khasraw, the Australian liaison for the study. "We want to fail early and fail often so we can rapidly discriminate between effective and ineffective therapies," says Dr Khasraw.

Glioblastoma is a malignant brain tumour that grows and spreads aggressively, overpowering healthy cells by consuming their space, blood and nutrients. About 1500 Australians are diagnosed with glioblastoma each year. It has one of the poorest of all cancer outcomes-just 10 per cent of cases survive five years from diagnosis-and no effective therapies have been developed for more than a decade, despite hundreds of clinical trials of new drugs and technologies.

The collaboration, known as GBM AGILE (an Adaptive, Global, Innovative Learning Environment) is a large international adaptive clinical trial-so-named because clinicians and researchers will adapt and change drugs, doses and other aspects of the trial based on early feedback and results, rather than waiting years to make conclusions.

Dr Khasraw says the new line of attack against glioblastoma can be summarised in three words-disrupt, cure and collaborate.

"We'll be disrupting many of the traditional methods used in clinical trials," he says. "We will be applying the latest methods of trial design in the hope that multiple treatments can be tested within the same trial protocol, to accelerate drug evaluation far beyond current research methods. That's because we want to learn as much as possible from every patient-about treatments, combinations of treatments, and biomarkers-and ultimately use that knowledge for effective treatment for all glioblastoma patients.

"Given that there have been no therapeutic breakthroughs to date we are completely focused on developing a cure for this deadly disease. To do that we need break down the silos of information that hinder rapid progress. This will be an enormous international collaboration, including a relatively large number of patients who will be enrolled into multiple arms of an adaptive trial using Bayesian statistics to assess efficacy of many new agents or treatment approaches at the same time. This will allow much faster assessment of new potentially useful therapies."

CTC's Professors John Simes and Val Gebski are also part of the group that developed the new platform.

John Simes told the media today that GBM AGILE would allow treatments to be adapted in response to how they were affecting patients worldwide. "We can assess a range of promising treatments and say 'well this is promising but this one is actually not,'" he said.

The trial is expected to start recruiting patients about the middle of 2016. 

13 November 2015