What do you think? Leave a respectful comment.

Why the FDA approved a controversial Alzheimer’s drug

The FDA on Monday approved the first new drug to treat Alzheimer's disease in nearly two decades. Federal health officials said it may help slow the brain-destroying disease's progression, but the approval goes against the agency's independent advisers who said the treatment wasn't effective in clinical trials. Pam Belluck, health reporter for The New York Times, joins Amna Nawaz to discuss.

Read the Full Transcript

  • Judy Woodruff:

    The FDA's approval of a new Alzheimer's drug today followed months of debate within the medical community about the agency's procedures, which, as Amna Nawaz reports, the announcement has done little to quiet.

  • Amna Nawaz:

    That's right, Judy.

    The approval was based on two clinical trials. One showed some improvement in patients who'd been given the drug aducanumab, but the other found no benefit. A number of practitioners said that was not good enough and fell short of FDA's usual standards for approval. And, in November, an outside advisory board recommended that FDA not approve the drug.

    But other medical professionals said the need was so great that any medication that offered promise was worth trying. The last treatment for Alzheimer's came on the market some 18 years ago.

    Pam Belluck is a health reporter for The New York Times. She has been following all these developments closely. And she joins me now.

    Pam, welcome back to the "NewsHour." Thanks for making the time.

    Let's start with the news of the approval today. Even with this disagreement over how effective drug is, the FDA still approved it. Why? What do we know about what they based that on?

  • Pam Belluck, The New York Times:

    Well, what they said was, they actually acknowledged that there was not clear evidence of clinical benefit, that there was some kind of murkiness in the evidence about whether this will actually help patients slow down their memory and thinking problems.

    But what they said is, there is some suggestion of that. And one thing that everybody on all sides agree that the drug does do is that it attacks a key protein in Alzheimer's disease. This is a protein called amyloid. And it is the protein that clumps into plaque in the brains of people who have Alzheimer's.

    So, they said, well, we are going to approve this drug because it does can attack the biology of the disease. We have some signal that there might be some benefit for patient symptoms, and we are going to tell the company that they need to do another clinical trial. And if the results of that trial do not show benefit, then the FDA might revoke the approval, and it could end up being that those trials don't show benefit at all.

    The issue with that is that the clinical trial takes three or four, five years. And so this drug will be on the market, patients will be using it without actually being certain that it has the ability to help them.

  • Amna Nawaz:

    So, while the drug is out there, I am assuming there will be high demand for it, right, with millions of people in the U.S. suffering from Alzheimer's.

    Do we know enough about the benefits and the risks at this point? What did they find in the trials in terms of the risks that people take on in taking this drug?

  • Pam Belluck:

    Yes, there is definitely a risk.

    This drug can cause brain swelling and brain bleeding. And in the trial, about 40 percent of patients in the trial did experience that. Now, it is not quite as serious as it sounds, because most of those cases didn't actually produce any symptoms for patients. But some number did.

    And about 6 percent of patients had to quit the trials because they — of the brain swelling or brain bleeding. So those can be serious side effects. And, as a result, anybody who takes this drug will have to have regular brain MRIs to check to see if their brain is experiencing anything like this.

    In terms of the benefit that they found — and, as you said, in the beginning, only one trial found any benefit. Another completely identical trial did not find any benefit. The benefit in the trial that did seem to be positive is actually quite slight.

    So, some of the scientists who were concerned and arguing against approval were saying, we don't have a slam-dunk here. We only have one trial that shows any possibility of benefit. And that benefit itself is pretty slight. And then, on the other side, we know that there is a risk. And so they were saying the potential benefit here doesn't outweigh the risk.

  • Amna Nawaz:

    Pam, here is the one of the question questions here, though, is that when the FDA issues approval, that implies this drug is safe, this is OK to use, and this is effective.

    There doesn't seem to be a clear-cut case for this here. So what is the role of the FDA in all of this?

  • Pam Belluck:

    Well, I think FDA is looking at the seriousness of this disease, the fact that millions of people have it, that there is very little on the market that actually helps for more than a few months.

    And they are saying that this drug seems to have a hint of benefit and it goes after this key protein of amyloid. And that is what they are sort of banking on. Now, there isn't a lot of really strong history with drugs that have tried to attack amyloid. There is about 25 years of failed clinical trials with anti-amyloid drugs.

    They have succeeded in knocking down the protein, in clearing it out of the brain, but they have not succeeded in producing any benefit that people would actually notice in terms of their symptoms.

    So, that is kind of the gamble that the FDA is taking here. They are saying, we think this one is going to be the one that sort of crosses that threshold. We think that its ability to get rid of amyloid in your brain is actually going to help you see that your memory loss declines at a slower rate.

  • Amna Nawaz:

    That is Pam Belluck of The New York Times joining us tonight.

    Thank you so much, Pam.

  • Pam Belluck:

    Thank you.

Listen to this Segment