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
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
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
The trial is expected to start recruiting patients about the
middle of 2016.
13 November 2015