A New Approach for a Deadly Brain Cancer

November 4, 2016, 3:41 PM UTC

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, crowdsourced 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 GBM AGILE here and here. And I’ll follow up on their progress soon.

More news below.


Next-gen biotechs are getting hit with a big dose of reality. The American Society of Hematology (ASH) annual meeting may be the single most important biopharma event of the year that's not the American Society of Clinical Oncology (ASCO) cancer conference. Yesterday, a whole stash of scientific abstracts for the event (which is in early December) was released—and it contained some very sobering news for closely-watched biotech firms like bluebird bio, Spark Therapeutics, and Achillion, whose share prices all got decimated in Thursday trading. Those first two firms are working on gene therapies, but Spark's abstract revealed adverse events for a patient in a small hemophilia B trial, while investors didn't consider the latest data for bluebird's Lentiglobin gene therapy for beta-thalassemia particularly impressive. (Endpoints)

Can "shocking" your pain away be an alternative to opioids? One of the biggest problems facing chronic and severe pain patients is that, even if they avoid addictive and potentially deadly opioid painkillers like Vicodin, their pain doesn't just disappear. That's why one startup, ENSO, is aiming to create a patch that uses electrical stimulation to ease pain. CEO Shaun Rahimi thought up the concept based on his own experience with chronic spine pain and a family friend who used the electrical shock method on him. (Fortune)

IBM thinks cognitive security will be available to any hospital in 3 years. Cyber attacks on health care institutions are becoming increasingly common, and that trend is only going to get worse. But hospitals can fight fire with fire, according to IBM researchers, by leveraging artificial intelligence and machine learning that can cut through a clutter of data to identify the most promising avenues for protection. "Cognitive security looks to unlock a new partnership between security analysts and their technology to analyze security trends and distill enormous volumes of structured and unstructured data into actionable knowledge," said IBM researchers in a new report, noting that surveyed security professionals were optimistic about the tech's potential. (Healthcare IT News)

A conversation with Shark Tank's Robert Herjavec. Today, I'll be chatting with Shark Tank investor and tech entrepreneur Robert Herjavec. We'll discuss his involvement in a competition seeking to find tech-based, non-drug solutions to help cancer patients. Stay tuned for a writeup of our conversation.


Feds reportedly set to file price fixing charges against more than a dozen generic drug giants... A bombshell report from Bloomberg on Thursday says that federal prosecutors are prepping antitrust suits against more than a dozen of the biggest generic drug makers in the world for collusion on drug price hikes by the end of the year. Companies like Endo, Mylan, Teva, Lannett, and others are among the firms being probed in the investigation, which has been going on for several years. The firms have denied any wrongdoing and pledged cooperation with the Justice Department; their stock prices fell sharply on the news. (Fortune)

...and lawmakers want the probe expanded to include diabetes treatments. But the generic drug companies aren't the only ones under siege for potentially questionable pricing habits. U.S. Senator Bernie Sanders and Rep. Elijah Cummings, the ranking Democrat on the House Oversight Committee, are calling on antitrust investigators to expand their probe to insulin makers like Sanofi, Eli Lilly, and Novo Nordisk. The pair of lawmakers has made scrutinizing drug price hikes one of its main missions in Congress and called for investigations into many of the pharma companies that will reportedly be charged in the Justice Department's antitrust suit several years ago. And insulin makers make sense as a target for more scrutiny; prices have been steadily rising, and the increases among competitors appear to occur in lock step at times. (Fortune)

Private firms flipping old drugs are contributing to price hikes. See a trend here? Drug pricing, which has become one of the most significant health care-related issues in 2016 not called "Obamacare," dominated pharma's news cycle this week and, alongside some of the discouraging biotech news from the ASH abstracts, has been an albatross around the sector's neck in multiple trading sessions. Bloomberg reports that private equity-backed firms have been snapping up older medications and then significantly hiking their prices, contributing to the treatment inflation that's been stoking public outrage. (Bloomberg)


The suicide rate among middle school students has doubled. Car crashes are now less of a death risk for American middle school students than suicide, according to new government data released Thursday. Part of that has to do with the overall decline in traffic fatalities; but the report also finds that the rate of suicide among these students doubled between 2007 and 2014. The reason behind the spike is unclear for now. (Fortune)

American medical care is expensive, even with Obamacare. The Affordable Care Act, aka Obamacare, provides financial assistance for individual insurance plan holders who use the law's marketplaces to counteract potential premium hikes. But the tradeoff for lower premiums in some cheaper plans is higher deductibles and out-of-pocket maximums, which can put a major financial strain on consumers who might need to use medical care. Of course, this phenomenon isn't limited to Obamacare; employers have also been relying more and more on high-deductible health plans in a bid to slash their own health-related spending and push more of the burden onto workers. (Bloomberg)

Soda taxes have big tests coming up in California and Colorado. The "War on Sugar" has several key upcoming battles on Election Day, when voters in California and Colorado will determine whether or not to levy taxes on sodas (as has been recommended by the World Health Organization). The campaigns have drawn opposition from beverage giants like Coca-Cola and grocery trade organizations. Earlier this year, Philadelphia passed its own soda tax. (Fortune)


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