This is the first new drug approved for Alzheimer's disease since 2003. It's the first to show significant progress against the sticky brain plaques that are the hallmark of Alzheimer's disease.

Transcript

MARY LOUISE KELLY, HOST:

Today the Food and Drug Administration approved a controversial drug to treat Alzheimer's disease. It's the first new treatment for the disease in nearly 20 years. It is also the first ever to treat the disease process underlying Alzheimer's. Joining us now to talk about the drug is NPR science correspondent Jon Hamilton.

Hey, Jon.

JON HAMILTON, BYLINE: Hi.

KELLY: Hey. So give us the basics. What is this drug? How does it work?

HAMILTON: Well, until today, this drug was called Aducanumab. It will be marketed under the brand name Aduhelm. And it's meant for people in the early stages of Alzheimer's, you know, before memory and thinking have been severely affected. What this drug does is to help remove some of the sticky amyloid plaques that tend to build up in the brains of people with Alzheimer's. It will be given as a monthly infusion, so directly into the bloodstream. And patients on Aduhelm will need periodic brain scans because the drug can cause swelling in the brain.

KELLY: I mentioned this drug is controversial. How come?

HAMILTON: Because it's still not entirely clear how effective this drug is. Here's David Rind of the Institute for Clinical and Economic Review.

DAVID RIND: It's very hard to know right now whether this drug works and, if it does work, how well it works and whether any benefits it might have outweigh its harm.

HAMILTON: So the FDA reviewed two big studies of this drug. One showed that it helped patients. The other showed that it didn't. And a panel of scientists who advise the FDA said the agency should require another study before approving this drug. The FDA decided to approve it anyway, but they said the drug's maker will need to conduct that confirmatory study after the drug is already being sold.

KELLY: So questions about whether it will actually work. Where does that leave patients? Is having this drug on the market a good thing for Alzheimer's patients and their families?

HAMILTON: Well, you know, so at this point, having the drug - you know, you have groups like the Alzheimer's Association who say it is a good thing. And here's Harry Johns. He's the association's CEO.

HARRY JOHNS: Today, unlike yesterday, there is hope for a treatment that could add additional cognitive and functional time for an individual and for their loved ones.

HAMILTON: Right. So the Alzheimer's Association pushed really hard to get this drug approved. And their position is that having even a marginally effective drug will give patients hope and maybe some benefit. Until today, the only drugs out there just treated the symptoms of Alzheimer's - you know, the memory loss and that sort of thing. They did not treat the underlying disease. This drug has at least the potential of slowing down the disease by reducing plaques in the brain.

KELLY: OK, so that's the case for approving this drug. What about the other side? What are the downsides?

HAMILTON: Well, one fear is that people will have unreasonable expectations that this is some kind of a cure. I mean, it's not. And also it may become more difficult to study other new Alzheimer's drugs now because people won't want to gamble on some unknown treatment when there is already one out there that has been approved by the FDA.

KELLY: And before we let you go, Jon, how much is this going to cost?

HAMILTON: A lot, probably. The company is talking about a base cost of around $56,000 a year. But this drug - it has to be given in a medical setting, and it requires periodic brain scans. So the total cost is likely to be a lot more. And insurers, including Medicare, aren't going to pay the whole cost. So patients and their families could end up spending thousands of dollars a year out of pocket.

KELLY: NPR's Jon Hamilton - thank you, Jon.

HAMILTON: Thank you.

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