Alzheimer’s is a debilitating disease that affects more than six million Americans. While there’s no cure, the U.S. Food and Drug Administration, earlier this month, approved a new treatment for the first time in 18 years. WUWF recently discussed the pros and cons of the new drug with an Alzheimer’s researcher from the University of West Florida.
“This is a tough one. The approval of aducanumab is a challenging one to wrap your head around, I think, in some ways,” said Dr. Rodney Guttmann, professor of biology in the UWF Hal Marcus College of Science and Engineering. He’s been researching Alzheimer’s disease for more than two decades.
“I understand why you would approve such a drug. There hasn’t been anything approved in 20 years and certainly, there is evidence of amyloid’s role in Alzheimer’s disease,” he said.
“The challenge has been that many clinical trials related to clearing the plaques have not shown much clinical benefit and that seems to be the case here with aducanumab, as well, that the amount of improvement is not that significant based on the clinical trial data.’
To be effective in treating Alzheimer’s, Dr. Guttmann says a drug has to go beyond the idea of just clearing plaques in the brain.
“The idea is that if you can clear the plaques people should get better, and by better I mean improved cognition, improving memory, improved behavior, less anxiety, better sleep; all the things that go along with Alzheimer’s,” Guttmann explained. “And that’s not really what was observed in the study. There was not a huge improvement in the cognitive outcome.”
The FDA approval of the drug, despite expert criticism that there’s not enough evidence to show it slows cognitive decline. The controversy led to the resignations of three advisory board members.
The process for approval is also a point of contention, in that the FDA stopped the clinical trials for aducanumab some time ago when it looked like the drug wasn’t going to work. Then the agency agreed to revisit incoming data that showed some effectiveness.
“Generally, if you fail to meet your primary endpoint, that would be the end of that. However, it seems as though, this is not quite falling into in that category,” said Guttmann, suggesting that many in the scientific community believe FDA changed the rules mid-game.
“I just think a lot of the community looks at this and says, ‘This is how we designed our clinical trials in the past.’ It didn’t work, we were required to stop and shift gears,” he explained. They (the company behind the new drug) stopped, then they got to revisit their data and now has FDA approval.
“I think that’s rubbing some people the wrong way,” said Guttmann. “And, it’s certainly a reasonable expectation. If you’re investing billions of dollars in your research and it doesn’t go and your drug is kind of the equivalent to the drug what’s here and now and they get to go, you can see how it would be problematic for some people.”
Other criticisms of aducanumab center on its side effects to include brain swelling and bleeding. Also, this is an expensive drug – to be delivered through infusion - that costs about $50,000 a year. Physicians will have to make judgments about eligibility and risk/benefit.
On the other hand, Dr. Guttmann recognizes why FDA approval of the drug is being heralded by organizations such as the Alzheimer’s Association, which sees it as the “beginning of a completely new future for Alzheimer’s treatments.”
Reinforcing that notion, he proclaimed, as he often does during presentations, that “science only moves in one direction.”
“We are getting closer every single day. We know more today than we did yesterday. And, in that regard, certainly, this drug is showing that the scientific community as a whole is pushing as hard as humanly possible to do something about this.”
And, because Alzheimer’s is a devastating disease that currently has no treatment or cure, this new drug believed to slow its progression represents a long-sought sense of optimism.
“Something out there that may be helpful certainly is a positive, in the sense that you know on the inside, in your heart. And, that’s the battle that we’re fighting right now is our heart verses. our head, right? The scientific data isn’t as strong as we’d like it to be, but definitely, there’s hope out there.”
Guttmann has contributed to efforts to find effective treatments for Alzheimer’s. The pandemic forced him to halt his most recent clinical trials. Drawing a parallel, he says scientists might be further along in development if they could recruit a fraction of the hundreds of thousands of people who participated in the COVID-19 vaccine trials.