Our Bold Response to the Deadliest of Brain Cancers

November 12, 2021

Guest Blog by Dr. Anna Barker, GBM AGILE Project Director

It’s long past time to act – the time is now! This year, tens of thousands of individuals around the globe will be diagnosed with glioblastoma (GBM) – the most common and deadliest of malignant adult brain tumors. It is cancerous and highly aggressive. This disease took the lives of Ted Kennedy, Beau Biden, and so many others over the course of this year and every year. Fifty percent of GBM patients will survive for a year or less, and the five-year survival rate is approximately 5.1% based on new CBTRUS data.

These dismal statistics have not changed for decades. They are mind numbing and frustrating, as there have been few real opportunities to change outcomes and improve survival – until now.

That’s why we are pleased to announce the GBM AGILE trial. This trial represents the first global initiative with an increased hope that these dismal survival statistics can be changed for the better. Created by a groundbreaking global coalition of some of the world’s leading GBM neuro-surgeons, neuro-oncologists, and clinical investigators and researchers, the GBM AGILE trial will be a learning system, where a patient’s individualized response to specific treatments will inform the ongoing direction of the trial.

I believe that through the advancement of GBM AGILE, we will identify new therapies for GBM if they are out there. Moreover, the adaptive design will enable us to move promising therapies (and combinations) more quickly and efficiently into late phase trials – and ultimately to patients. We also hope to more accurately identify predictive GBM biomarkers that will ultimately ensure more effective treatments.

AGILE stands for Adaptive, Global, Innovative Learning Environment – and it will be an unprecedented, and likely the largest and potentially most important, global collaboration in the history of brain cancer research. Unlike traditional clinical trials that test each intervention using defined study protocols held constant throughout the trial, GBM AGILE will be an adaptive, continuous learning system whose outcomes are analyzed constantly to inform the trial’s next steps. Its flexibility in design will help us modify the trial as it proceeds based on real patient data – addressing uncertainty about original design choices – and will allow for multiple new drug combinations and treatments to be used while dropping ineffective drugs quickly.

I believe that adaptive trial design is one of those innovative approaches that represent the future of precision medicine – and GBM AGILE embodies this “state of the art” paradigm in all respects.  Adaptive design is rapidly gaining interest and support with researchers and drug companies because it can more quickly help identify drugs and biologics that do and don’t work, and find biomarkers that can be used to enroll more patients in later stage studies.

Changes, revisions and new directions can be planned and executed more quickly through an adaptive trial like GBM AGILE, with potentially improved treatment plans and outcomes. This trial will finally enable us to bring precision medicine to this most aggressive and deadly brain tumor – and that is good news for patients. 

Adaptive design also can impact costs while creating an environment for more successful late-stage trials. We will aim to drastically reduce development time that will help reduce the costs and time of future drug development. A trial like GBM AGILE can take a lot of time off the clock and save millions of dollars in costs, while potentially seeking FDA approval earlier through smaller focused less expensive phase 3 trials, to ensure treatments reach patients more rapidly. 

Our coalition is truly committed to the work ahead. The efforts needed to design and implement GBM AGILE are well underway. Currently, more than 130 researchers, neurosurgeons, imagers, and patient advocacy organizations compromise ten major committees that donate their time and pay their own travel costs to participate in the design and planning process. We are “crowdsourcing” knowledge from a large number of international leaders in basic and clinical research.

We meet regularly, despite our many time zone differences, and tackle challenges related to everything from drug selection, to biomarkers and other design issues. None of us are willing to continue to tolerate the tragic and costly loss of life inflicted on patients who are stricken with GBM. GBM AGILE is truly a global coalition of the willing, and it’s always humbling to see the power of a group this committed to changing the world.

Our hope is that GBM AGILE will eventually provide an opportunity for patients to benefit from the type of precision medicine that is tailored to each and every patient. We hope for new therapies as opposed to the status quo of many failed clinical trials and only one new unimpressive treatment in the last decade. Our hope is that GBM AGILE can be a source of optimism for GBM patients and their families, as our ultimate goal is to significantly improve the quality of life and survival rates of the many who are afflicted with GBM. Stay tuned for updates and more information next year.