How Mouse Studies Lead Medical Research Down Dead Ends : Shots - Health News New drugs are usually tested in animals before they're tested in humans. But many of those studies aren't done carefully enough, analysts say. So time and money is wasted, and treatments are delayed.
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How Mouse Studies Lead Medical Research Down Dead Ends

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How Mouse Studies Lead Medical Research Down Dead Ends

How Mouse Studies Lead Medical Research Down Dead Ends

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Searching for an effective drug can take a decade. It involves careful work, millions of dollars. Many drugs that look promising simply don't pan out. But researchers say many of those failures can be caught earlier in the testing process, saving precious money and time.

Here's NPR's Richard Harris.

RICHARD HARRIS, BYLINE: To get a sense of this problem, consider drugs that are being developed to treat ALS, or Lou Gehrig's disease. In the past decade or so, nine potential drugs have been tested in people who have this degenerative nerve disorder. Not one has been effective. So Steve Perrin, who runs the ALS Therapy Development Institute in Cambridge, Mass., decided to take a close look at the mouse studies that had initially suggested these drugs were promising.

STEVE PERRIN: We tried to replicate those findings precisely by talking to the authors and trying to repeat the experiments in an identical fashion. And what we found is that we couldn't replicate a lot of the experiments.

HARRIS: Perrin says that if scientists had been more careful with their initial mouse studies, they would have realized that these drugs were never good candidates, so it made no sense to try them in people. And he writes in Nature that scientists who did those early studies would have known that, if they had done those studies correctly. Instead, it ended up wasting a huge amount of time and money.

PERRIN: Once you start getting into clinical trials, you're talking hundreds of millions of dollars per drug.

HARRIS: It's also a rough deal for the patients who have diseases like ALS, which can progress rapidly, if they choose to try an experimental treatment.

PERRIN: Patients have one shot on goal. If it doesn't work for them, they've lost that one shot. They might not qualify for the next trial that looks promising, or they may lose their battle with the disease.

HARRIS: And this is not just a problem for ALS. It's true for cancer research as well. Glenn Begley worked at the drug company Amgen to develop cancer drugs. He also noticed a lot of faulty animal studies in the early stages of drug development. He decided to review more than 50 animal studies that had come across the transom, to see how many of them were valid.

GLENN BEGLEY: And I was, frankly, shocked to find that the number was more like 90 percent of papers that we were unable to reproduce.

HARRIS: Because Amgen and other big pharmaceutical companies know that many animal studies are dubious, they always re-do them before deciding whether to go ahead with human studies. So the company did not end up wasting hundreds of millions of dollars, or years, on a dead end.

BEGLEY: For small companies that have got limited resources, they probably do go ahead with studies that ultimately will burn out.

HARRIS: Begley says this problem doesn't apply to drugs that make it all the way through the approval process. But these early errors clearly slow the search for new drugs. Begley, who is now at Tetralogic Pharmaceuticals in Pennsylvania, says it's not that scientists doing these tests are deliberately sloppy.

BEGLEY: Fundamentally, it's the way we as human beings operate. We really want results to be positive. We really want to discover something that's actually going to improve human health.

HARRIS: So scientists are too eager to seize on hopeful results, and not skeptical enough about their own work. Drug studies in human beings take that into account by making sure that scientists running the tests don't know who's getting the drug, and who's getting the comparison pill. Begley says that same standard should be applied to animal studies.

Perrin at the ALS Therapy Institute says, yes it will cost more in the short run to do it right, but it will avoid wasting time and money in the long run.

We can't possibly keep living under the same system that we have been for the last decade or so.

Scientists doing this research need to be rewarded for getting it right, Begley says, not just getting it published.

Richard Harris, NPR News.


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