The current approach to clinical trials is outdated.
“The standard clinical trial is pretty much the only thing in medicine that hasn't changed in the last 70 years,” says Don Berry, a revered biostatistician at M.D. Anderson Cancer Center in Houston, who has been working to modernize the drug-testing method for at least half of that time period. Berry, along with Anna Barker—a former deputy director of the National Cancer Institute and now a professor at Arizona State University—and others have long pushed for a trial design that lets investigators ask lots of questions (not just one), test multiple drugs at the same time, and most importantly, learn as they go.
And now, this revolutionary approach is being put to the test, with sanction from the FDA, in one of the deadliest cancers around: glioblastoma multiforme, or GBM—an aggressive brain cancer diagnosed in more than 12,000 Americans each year. The new trial, called GBM AGILE (the acronym stands for Adaptive Global Innovative Learning Environment), will test several experimental agents and established drugs on patients, divided into subgroups on the basis of individual biomarkers. The drugs that have no effect will be quickly dropped as others—in some cases, “crowd-sourced” from more than 100 experts around the world—are subbed in. All the while, researchers will study the responses in each patient and try to learn from them, incorporating whatever insights they can glean into the evolving trial. (In traditional clinical studies, by contrast, investigators typically wait until the end to see what happened.)
