Glioblastoma Multiforme (GBM) is a devastating disease. The deadliest and most aggressive form of brain tumor, GBM is expected to claim the lives of 12,000 people in the United States this year, plus tens of thousands more around the globe. For these patients, GBM is currently a death sentence: for every 1,000 patients diagnosed, fewer than 500 survive a year after diagnosis; after five years fewer than 20 will remain. These statistics have remained unchanged for decades.

For us at the National Foundation for Cancer Research (NFCR), this situation is unacceptable. Out of determination to do something innovative and new to change this status quo, we joined an unprecedented international coalition to launch GBM AGILE – a global effort to defeat GBM through next-generation clinical trials. This is truly a global coalition, with over 150 participants from more than 40 leading cancer institutions across four continents.

At the heart of GBM AGILE (which stands for Adaptive Global Innovative Learning Environment) is the concept of the “adaptive” clinical trial. Standard clinical trials take 3-7 years to produce results, and they cannot be modified once they begin. In this model, patients get only one opportunity, receive only one treatment, and by the time results are produced the treatment protocol is already many years old. By contrast, adaptive trials test multiple treatments and combinations of treatments in parallel, at the same time. Adaptive trials are also designed to be continuously updated, to incorporate the latest information using a technique called Bayesian Statistics. This feature provides the “Innovative Learning Environment,” in which ineffective treatments can be shut down early, and new treatments can be initiated quickly.

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