The Food and Drug Administration’s approval of the first new drug treatment for Alzheimer’s disease in nearly 20 years has drawn mixed reactions from the medical field nationwide.
While some who work in the Alzheimer’s field celebrated the approval of Aducanumab, critics called the nod for the drug reckless, pointing out there is not sufficient evidence that the drug is effective.
For years, Alzheimer’s patients had few options for the mind-robbing disease. Patients only used prescribed medications to treat underlying symptoms like anxiety and insomnia. Now, Aducanumaba is a drug designed to get to the root of the problem in Alzheimer’s patients, growing plaques in the brain which hinder cognitive function.
“Aducanumab, it is an antibody which recognizes amyloid, which is a substance that accumulates in the brain in Alzheimer’s disease,” said Dr. Marsel Mesulam, director of the Mesulam Center for Cognitive Neurology and Alzheimer’s Disease, Northwestern Medicine. “So Aducanumab binds to the amyloid and removes it from the brain that is its function.”
The drug maker, Biogen, says it works like detergent to remove dirt from the brain. But in two major trials, results were conflicting.
“In one of them, it seems the results were clearly negative,” Dr. Mesulam said. “In the second one, it was trending toward positive.”
Moreover, an FDA advisory panel initially recommended rejecting the drug. But as more data trickled in, showing the drug worked in the brain, the drug, administered as a monthly IV infusion, was given the nod.
The big question now is will it help patients day-to-day?
“I think it is important to point out that the drug did exactly what it is supposed to do in the brain. It removed amyloid. We have evidence for that,” Dr. Mesulam said. “The issue is whether or not it has any clinical efficacy and even the most lenient interpretation of the data shows an improvement of about 23%, which is very small. It may not be perceptible clinically, but of course, 23% is better than zero.”
That’s what the Alzheimer’s Association is clinging to — improvement that makes a difference, even if it is small.
“Something that could help slow down progression of the disease and that is what we’re excited about as a science-based patient advocate organization,” said Delia Jervier, executive director of the Illinois chapter Alzheimer’s Association. “We’re excited about anything that could provide relief to families.”
Cautious physicians say in addition to having a choice for patients, the approval could have another advantage.
“I think the important benefit might be psychological,” Jervier said. “That it may create this kind of hope and momentum in the field that progress speeds up.”
With hope, physicians will now prescribe the drug in early-stage patients to prevent damage. The new drug does not reverse mental decline, however, and all new users will be monitored.
“What happens is now that the FDA has taken a chance on this drug,” Jervier said. “There is still going to be a post-approval evaluation. So we hope that the increased number of patients who are going to be tried on this drug will surprise us on the upside and that, in fact, the drug will prove itself to be more useful than the available statistics show. So that would be great.”
What’s not so great is the price. The drug could cost between $30,000 to $50,000 per year. Potentially serious side effects include brain swelling and bleeding. Officials in the medical field say if the new data does not show improvement for patients, the FDA could pull the drug from the market.