Why GBM AGILE?

While there have been innovative and cutting-edge advancements in biomedical research, GBM treatment options and patient outcomes have remained largely unchanged over several decades. In fact, there is currently no known cure for glioblastoma (U.S. Department of Health & Human Services).

Despite previous efforts to improve patient prognosis, GBM kills 95% of patients within five years of diagnosis, with more than half dying within the first 15 months after diagnosis (National Foundation for Cancer Research). Although first-line treatment has been clearly defined since 2005, no standard second-line treatment has yet been determined (Current Oncology, “Nonsurgical treatment of recurrent glioblastoma”).

Our goal is to solve this dilemma by operationalizing an adaptive platform trial called GBM AGILE.

 

What is GBM AGILE?

Building on previous biomedical research discoveries, including traditional randomized controlled trials, a committed group of over 130 oncologists, statisticians, pathologists, neurosurgeons, imagers, advocates, and researchers from academia, industry and government have been working collectively since 2015 to design a new type of randomized clinical trial – GBM AGILE – that will further enhance the way therapies are identified for this deadly disease. This remarkable “knowledge network” of global experts, led by the GBM AGILE Executive Committee, met regularly as collaborative teams to shape the adaptive design and master protocol for GBM AGILE. The Cure Brain Cancer Foundation, National Foundation for Cancer Research, National Biomarker Development Alliance, and National Brain Tumor Society played a major role in funding and development of GBM AGILE, along with experts from the United States, Australia, Canada, China, Germany and elsewhere.

GBM AGILE is an entirely unique approach to clinical trials made possible by the partnership and collaboration of clinicians, researchers, governments, regulatory agencies, pharmaceutical companies, and patient advocacy groups.

GBM AGILE

How is GBM AGILE different from other clinical trials?

Traditional randomized controlled trials (“RCTs”), while scientifically rigorous and considered to be the ‘gold standard’ in clinical research, are limited in many ways:

GBM AGILE is a flexible and adaptable trial approach that maintains several “treatment arms,” giving it the potential to fundamentally change the clinical research process for GBM by identifying or disproving therapies more quickly. This will help to usher in a new generation of effective treatments for GBM, the most common and the deadliest type of brain cancer accounting for 45% of all malignant brain tumors (National Foundation for Cancer Research).

This new clinical trial approach is advantageous to GBM patients because it will help to identify effective treatments faster, allowing researchers to simultaneously and dynamically study the effects of multiple unique drugs.

GBM AGILE uses adaptive randomization allowing for dynamic adjustment. If one treatment arm outperforms another, a higher proportion of new enrollees will be assigned to that treatment arm, a technique called ‘adaptive randomization.’ Simply put, GBM AGILE is patient-centric (not drug-centric) and provides a streamlined method for researchers to utilize data connectivity within the trial to answer many questions concurrently.

 

THE CLINICAL TRIAL OF THE FUTURE

AS ITS FIRST PRIORITY, GCAR IS THE OFFICIAL SPONSOR OF GBM AGILE, AN ADAPTIVE PLATFORM TRIAL FOR PATIENTS WITH GLIOBLASTOMA – THE DEADLIEST FORM OF BRAIN CANCER

GBM AGILE
 

Each year, more than 12,000 people living in the U.S. and tens of thousands more worldwide will receive a diagnosis of GBM (National Foundation for Cancer Research). GBM AGILE has the potential to substantially reduce development time for new therapies and biomarker discovery. This increased efficiency generates tremendous value for sponsors seeking new drug approval or new indications for previously approved drugs.

GBM AGILE will transform clinical care into an optimized learning environment where patients have access to the latest, most personalized therapies on a global scale.

In addition to the inherent value of faster therapeutic development for patients with GBM, GCAR would seek to leverage the value creation from GBM AGILE to focus resources on underfunded areas of promise for GBM patients.

 

Who is Eligible for GBM AGILE?

GBM AGILE is enrolling both newly diagnosed and recurrent GBM patients to evaluate which therapies work best for each type and biomarker subtypes. The trial will take place across multiple clinical sites (e.g., university research centers, hospitals, and cancer institutes) throughout the United States, Canada, Australia and elsewhere.

 

Strategic Partners