FDA Advisory Panel Rejects Controversial Alzheimer's Drug
MARY LOUISE KELLY, HOST:
In other news, hopes for an experimental drug for Alzheimer's disease took a big hit today. It failed to win support from a panel of experts who advise the Food and Drug Administration. NPR science correspondent Jon Hamilton is on the line.
JON HAMILTON, BYLINE: Hi.
KELLY: So what is this drug that failed to win support from our panel of experts?
HAMILTON: It's called aducanumab, and it's made by Biogen. And it's designed to go into the brain and clear out those sticky amyloid plaques that are associated with Alzheimer's. The studies have shown that it's really good at getting rid of those plaques. And aducanumab has gotten a lot of attention because if it were to be approved, it would be different than any existing drug for Alzheimer's. It would actually treat the underlying disease process that kills brain cells instead of just reducing symptoms.
KELLY: So why is this panel not supporting it?
HAMILTON: Well, today's meeting was sort of a tale of two big studies and a lot of drama. One of these studies showed that aducanumab slowed down the progress of Alzheimer's. The other showed that it didn't. Usually, you need two big studies finding the same thing to approve a drug. But at today's meeting, the FDA asked its experts to focus only on the positive study, and it also presented analysis of the data that was really very favorable to the drug. All of that did not go over well with these advisers. Some of them seemed to feel like they were being railroaded by the FDA. Here is Scott Emerson, Dr. Scott Emerson. He's a biostatistician at the University of Washington.
SCOTT EMERSON: This analysis seems to be subject to the Texas sharpshooter fallacy, a name for the joke of someone first firing a shotgun at a barn and then painting a target around the bullet holes.
KELLY: Wow. OK, so they really don't like the process.
KELLY: But, I mean, does the drug work? Do we know?
HAMILTON: Nobody seems to be sure. The advisers seem very uncomfortable with making a decision about this drug based on the data they had. Also, there are a number of prominent scientists who submitted comments to the FDA who are even more skeptical. They didn't say it didn't work, but they said that we don't have the evidence to show that it does. I talked to Dr. Lon Schneider, who directs the California Alzheimer's Disease Center at USC. And I asked him flat out, should the FDA approve aducanumab?
LON SCHNEIDER: No. One identical study was flat-out negative. The other one was biased and unblinded, and whatever effects it showed were truly trivial.
KELLY: Among the questions about any possible new drug, John, there's, does it work? There's also the side effects. Do we know any dangers to this drug?
HAMILTON: There are some. A fair number of people who take it experience this swelling in the brain. And to be fair, it seems to go away when they stop taking the drug and doesn't seem to cause long-term problems. But like with most drugs, you know, there is some risk.
KELLY: Well, so what comes next? Is this the end of the road, or do we know what the FDA will do?
HAMILTON: It is really unclear. I mean, usually the FDA follows the recommendations of its advisory panels. In this case, though, the agency has all but said it really, really wants to approve this drug. So even though they got a lot of pushback from the advisory panel, it's still possible that the FDA will approve aducanumab. And if that happens, the concern is that potentially millions of people could receive a drug that doesn't help them, and it might hurt them.
KELLY: NPR's Jon Hamilton reporting there.
Thank you, Jon.
HAMILTON: My pleasure.
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