News & Blog

GBM AGILE: A Community Galvanized

“GBM is just unacceptable…we no longer accept the status quo.” – Dr. Anna Barker

Last week, you likely took notice of the press release, guest blog from Dr. Anna Barker, and a flurry of social media activity related to the announcement of the GBM AGILE coalition and clinical trial.

As NBTS is co-chairing the GBM Advocates Committee of the coalition and trial, as well as co-sponsored and participated in the early planning meetings that led to the development of GBM AGILE, I had the great privilege of attending the kick-off event in person along with our interim CEO, David Arons.

The kick-off, taking place appropriately in our nation’s capital, consisted of a press conference at the National Press Club followed by a reception for all of the researchers, clinicians, and advocacy organizations taking part.

“GBM is an insidious foe, fully capable of rewiring its circuitry to evade drugs.” – Dr. Webster Cavenee

The excitement was palpable at both events, as every participant realized the historic nature of the announcement that was being made and the great potential for impact the GBM AGILE trial could have for a patient population that is desperately seeking progress.

image8“This represents a sea change for this disease,” said project director, Dr. Anna Barker of Arizona State University, summing up the enthusiasm in the opening remarks of the day.

Moreover, it was amazing to see so many brain cancer experts from all over the world in attendance, including large contingents from participating nation’s China and Australia. Equally as impressive was the slate of guests on hand for the announcement, including representatives from biopharmaceutical companies, politicians, journalists, and patients and caregivers.

GBM AGILE Announcement

The four main themes from the announcement were that GBM AGILE, would: 1) be a completely new approach to treatment development and evaluation in brain cancer, 2) put patient needs at the forefront, 3) rapidly accelerate progress, and 4) serve as a harbinger and model for the future for not just brain cancer, but potentially other tumor types, as well.

Stressing the first point, Dr. Janet Woodcock of the U.S. Food and Drug Administration offered her perspective as the head of the center at the FDA that evaluates and approves drugs for all disease.

“This is the future we are seeing,” said Dr. Woodcock. “I think this is fabulous and is a tipping point for moving forward.”image7

Dr. Woodcock went on to express that the FDA supported this new approach to clinical trials, and that if GBM AGILE works (and we’re confident it will!), it would be the future not just for brain cancer, but for all clinical trials.

“To conquer GBM, we really have to do something different…This is going to turn the system inside out,” added Dr. Web Cavenee of Ludwig Cancer Research.

Dr. Donald Berry of MD Anderson Cancer Center, co-principal investigator for GBM AGILE and architect of the famous I-SPY trials in breast cancer, explained further the need for innovation.

“Randomized clinical trials [the current standard clinical trials] are 70 years old…what other technology doesn’t change in 70 years?” asked Dr. Berry. “Meanwhile, cancer biology is moving at light speed and potential treatments have to wait in the queue. GBM AGILE is different. It’s the first to merge clinical research & clinical practice.”

Dr. Amy Heimberger of MD Anderson Cancer Center explained that the improvements in trial design would also help put patients first in GBM AGILE.

“This is about the best thing for patients,” said Dr. Heimberger, noting that treatments being evaluated in the trial will be based on advance methods that make sure the drugs with the best chances of succeeding are given priority.

“If we can deal with GBM, we can deal with just about anything…GBM AGILE has the ability to enable progress in a number of diseases” – Dr. Barker

Dr. Berry concurred, and said he believed GBM AGILE, “has many advantages for patients.”

This includes speed. And as Dr. Woodcock put bluntly, “ We need answers more quickly.”

“We maximize the probability of success, and minimize the time it takes to get there,” surmised Dr. Berry.

image6Dr. Berry’s other co-primary investigator, Dr. Timothy Cloughesy of UCLA made clear that because GBM AGILE uses an innovative statistical approach to data analysis, known as Bayesian statistics, the trial benefited from a greater ability to predict treatment responses.

“Bayesian approaches are efficient and increase the likelihood of beneficial therapy,” said Dr. Cloughesy.

It is the deployment of Bayesian statistics which gives GBM AGILE its flexibility and allows it to work as an “adaptive” trial that learns from every patient and adjusts on the fly to get the right treatments to the right patients.

“If we do our work right – build, adapt, and learn – the impact of GBM AGILE will be huge,” concluded Dr. Cloughesy.

GBM AGILE Reception

Following the announcement was the reception for all the members of the GBM AGILE coalition, which was highlighted by a visit from Vice President Joe Biden.

image4The vice president, as you know, lost his beloved son Beau to brain cancer this past spring. And recently, Biden made an announcement that he would commit the rest of his time in office to fighting cancer. He showed his dedication by delivering an impassioned speech to the coalition members, reminding everyone in the room of the high-stakes.

The vice president – flanked by his son Hunter and daughter-in-law Kathleen – spoke of Beau’s personal battle and the strength and integrity he showed throughout, as well as the challenges his family has faced through the grieving process and trying to understand how to move forward. In that regard, he offered his words of support for the GBM AGILE effort.image2

“You helped me clarify in my mind what had to be done,” said Biden to GBM AGILE coalition. “You have helped me begin to realize how to get my arms around this…there is no reason why we can’t make gigantic strides.”

Vice President Biden went on to state that he believed that an effort like GBM AGILE was important because it could, “demonstrate to the American people that there is nothing we can’t do,” which is the goal of the “moonshot” in cancer he mentioned in his speech just a few short weeks prior. Particularly he pronounced how impressed he was to see the high-level of collaboration and that the community had united for this major effort.

“The cumulative capacity of brilliance in this room is awe inspiring to us,” he affirmed.

While all of us gathered in the room were thrilled to hear the vice president’s words of support and encouragement, it was truly his memories of Beau, and the strength that his family has displayed, that was inspiring.

NBTS and the rest of the coalition will use this inspiration, and the inspiration we receive every day from ALL of the brain tumor community, to continue to lead the fight through transformative and impactful projects, like GBM AGILE, that promise to make great leaps forward for patients.

Further, we are excited to see the synergies and mutual benefits between GBM AGILE, and our other cornerstone project, the Defeat GBM Research Collaborative. Defeat GBM was always designed to feed into a “fourth core” that would be a smart – or adaptive – clinical trial. We believe AGILE can be that trial. Many of AGILE’s leaders are the heads of project teams and/or on the Strategic Scientific Advisory Council for Defeat GBM (Drs. Web Cavenee, Anna Barker, Tim Cloughesy, Paul Mischel, and Al Yung) and it is our hope that their work as part of Defeat GBM will only help to inform the AGILE trial to contribute to its success.

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  • Richard Powers says:

    I lost my wife to a GBM and now one of my children has a Stage III anaplastic astrocytoma. I would like to hear more about the AGILE program. Is it a program that determines whether trials are worth funding by NIH or by the conductor of the trial, e.g., MD Anderson Hospital.? I would like to be inspired like Joe Biden, but I have insufficient information.

    • TomHalkinNBTS says:

      Thank you for writing, Richard. We are sorry to hear how this awful disease has impacted your family. As far GBM AGILE goes, it is itself a clinical trial. It is an innovative style of clinical trial that works across institutions, and tests multiple potential new treatments at once, while using the specific molecular ake-up of each person’s tumor to match them into subgroups most likely to benefit from the different treatments. You can learn more about GBM AGILE here:

      And more about Adaptive Trial Designs here:

  • Victor Buchholz says:

    I am very interested to hear and learn about this (to me) new ww . I come from Europe/Gy and my wife was successfully operated by AC (Awake Craniotomy) surgery at Heidelberg Univ 4 months ago. Her tumor ‘disappeared’ it seems…, as they say from looking at her last MRT pic. We are optimistic but also realistic. Currently we are into the ‘typical Standard RC/TZM mode’ for GBM treatment but nobody had dropped a word about anything like GBM Agile… As we have links to American and French friends, we had been advised on alternative treatment directions, be it towards ‘personalized precision drug medication’ or ‘immunotherapy or gene therapy or a viral therapy’. So, I had asked about a DNA Sequencing for my wife’s tumor specific analysis. The answer was ‘not at this – primary – stage’…so what and when? It seems to me as stated quite often, there is no real helpfull coordination beyond the first surgery and biopsy…Can you help me to find the entering point to your German cooperators.
    Thanks a lot in advance.

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