A New Approach for a Deadly Brain Cancer

Yesterday I wrote about the mindset of problem-solving that seemed omnipresent at Fortune’s Brainstorm Health. Some of that Fix-It-Ourselves worldview revolves around our ability, thanks to a flood of new technology, to make things we could never make before.

But importantly, much of the fixing needed in today’s healthcare culture involves changing systems and practices. That was clear from a lively lunchtime discussion on reinventing clinical trials. This vetting process, without question, helped turn the world’s pharmaceutical bazaar from one of snake oils and patent medicines to one in which every pill and potion in our medicine cabinet has been tested and evaluated to some degree. But it’s also lengthy, expensive, and inefficient. Too often, it takes so long that medical science itself has whizzed past it, making the question being asked—Is this particular drug better than the standard of care?—no longer relevant.

“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.)

It’s a trial design that has caught up, at last, to the age of precision medicine. You can read more about the GBM AGILE here and here. And I’ll follow up on their progress soon.