I was waiting in the doctor's office lobby yesterday and an article in the June 15 Newsweek (hey, that's more current than most lobbies) by Sharon Begley caught my eye. Begley is the magazine's science guru, on par with Bernadine Healy at US News and Sanjay Gupta at Time/CNN. I don't really care for any of them, but I suppose they serve their purpose of letting the weekly news magazines' readers feel they've kept up to date on science after reading a one page glossy on some topic that blurs the health/science boundary.
Back to Begley. Her article, "Academia slows the search for cures," takes the somewhat surprising approach of targeting not pharma but academia for impeding bench to bedside research (she mainly takes shots at NIH). Also surprising - I agreed with portions of her article.
Had I not seen today's Wall Street Journal, I may have let the article drift into the cobwebbed corners of my memory. The article, "Brain-Trauma Study Set," begins with the following:
Several potential treatments for brain injury and stroke have failed in recent clinical studies, but one improbable therapy -- the hormone progesterone -- continues to show promise in warding off brain damage from head trauma and stroke.Wait a minute. The little hamster that does the filing in my head pulled out the Begley article, and I was right - progesterone isn't improbable or recent at all. Begley, in the middle of a rant about why pharma doesn't invest in academia (which is another issue I took with her article, but more on that later), says:
In the latest development, the National Institutes of Health are expected Tuesday to begin funding a study evaluating the hormone in more than 1,100 emergency patients with moderate to severe head trauma.
This study will get under way at 17 hospitals in 15 states around the U.S. and is expected to last up to five years at a projected cost of up to $28 million. It will be the pivotal study of whether the naturally occurring hormone progesterone, injected into patients within hours of severe accidents, can lower deaths and reduce paralysis and cognitive damage.
But when a promising discovery has the profit potential of Pets.com, patients can lose out.A stark example is the work of Donald Stein, now at Emory University, who in the 1960s noticed that female rats recovered from head and brain injuries more quickly and completely than male rats. He hypothesized that the pregnancy hormone progesterone might be the reason. But progesterone is not easily patentable. Nature already owns the patent, as it were, so industry took a pass. "Pharma didn't see a profit potential, so our only hope was to get NIH to fund the large-scale clinical trials," says Stein. Unfortunately, he had little luck getting NIH support for his work (more on that later) until 2001, when he received $2.2 million for early human research, and in October a large trial testing progesterone on thousands of patients with brain injuries will be launched at 17 medical centers. For those of you keeping score at home, that would be 40 years after Stein made his serendipitous discovery.Hmm. We've known about progesterone and brain injuries for 40 years, but because there wasn't a profit potential it hasn't been explored? That doesn't surprise me - see every third world disease category, for example - but why didn't WSJ's Thomas Burton frame his story with that context? And why is Begley attacking academia in her article, but instead blasts pharma as the reason progesterone wasn't picked up? I'm confused. I kept reading the WSJ article, and the very next line was:
It will be the definitive test of earlier animal research conducted over a quarter century by Emory University brain researcher Donald G.Stein, the subject of a page-one Wall Street Journal article two years ago.Ok, that's a little better, but what happened to the bit about 40 years ago? And what's with the line about being the subject of a page-one WSJ article two years ago? That's hardly newsworthy, or relevant.
Burton has the chance to go into the history of this research later, but again just goes with the "decades" cover-all and the article still retains its "new news" feel. "The notion that it could be helpful against brain injury arose as an accidental finding," Burton writes. Yes - in the 1960s. Where is the outrage? Where is the call to action? Why is something that is potentially life-saving relegated to backwater benches until pharma or the government take an interest?
We wouldn't want that, of course. You need standard of care in trials like this. But what if the standard treatment could have been vastly improved a long time ago? And why the hell did it take 40 years to get something that occurs naturally into clinical trials, over the billions spent on synthetic failures? I know why - profit. But why blame academia? Why not put the blame on everyone who has deep enough pockets to support research, but doesn't because it doesn't have a good enough RoI? And why in the world is saving a life a for-profit enterprise?
"You can give progesterone up to six hours after the stroke and still get significant benefits," said Dr. Stein. This is animal research, but it is consistent with his lab's findings over decades.In the upcoming brain-trauma trial, patients will be randomly assigned to receive progesterone plus standard therapy to stabilize head injuries, or will be part of a control group receiving standard therapy alone. One potentially controversial aspect of the study is that it will be given to patients without consent, largely because time is of the essence and patients' families often can't be located for hours. No patient will have any standard treatment withheld, however.
The six-hour window could prove especially important in stroke, since the main treatment now for the condition, a drug called tPA, has so far been definitively shown to be effective for only a three-hour window after a stroke.