What a doctor who has tested new Alzheimer's drug wants you to know

June 29, 2021
Illustration of what Alzheimer's disease does to the brain.
Alzheimer's causes neuronal death and formation of neurofibrillary tangles and beta-amyloid plaques. Image by 7mike5000 from "Inside the Brain: Unraveling the Mystery of Alzheimer's Disease."

A dementia specialist at the Medical University of South Carolina involved in clinical trials testing the new Alzheimer’s drug, aducanumab, said some of his patients have been really happy with it. “They felt that it worked and slowed down their decline.”

But Nicholas Milano, M.D., said it needs further study. “I think it’s important to confirm whether or not this really works.”

The Food and Drug Administration recently gave aducanumab accelerated pathway approval. It does that when there’s a potentially valuable treatment for a serious disease, giving people access to the drug while it undergoes more testing. Aducanumab will be sold under the brand name Aduhelm.

Dr. Nicholas Milano 
Dr. Nicholas Milano

There’s a huge need for treatments for people with Alzheimer’s disease. It’s the sixth leading cause of death in adults in the United States.

“It’s a progressive disease with no cure that eventually will cause the end of someone's life. Initially it just affects memory, but then over time it progresses to other aspects of brain function. So it can affect language, problem solving, spatial skills and then eventually it affects motor skills as well. When you get to the severe stages, people have significant issues with not just their thinking, but also their mobility,” Milano said.

Aduhelm is the first new treatment for Alzheimer’s since 2003. It’s designed to slow the progression of the disease. “This drug is exciting because it works with the mechanism that we think causes Alzheimer's, which is this buildup of amyloid plaques in the brain, and it clears them out. So it logically makes sense, and hopefully it will work,” Milano said.

“On the other hand, there have been other drugs which had the same mechanism that did not show benefits.”

And Aduhelm is controversial. A panel of experts recommended against FDA approval last year because they didn’t think there was enough evidence that it worked.

Milano said there are also some risks for patients. “There’s about a 30% chance to have some brain swelling associated with it. The good news is most cases are asymptomatic or have very mild symptoms and you don't even need to stop the medication. But in some cases it can be severe.”

Milano, an associate professor in the College of Medicine, is involved in ongoing research in MUSC Health’s Memory Disorders Clinic, testing Aduhelm to see if it’s safe in the long term and works as well as some of his patients believe.

But the Alzheimer’s Association isn’t waiting to celebrate. It called the drug’s FDA approval “a victory for people living with Alzheimer’s and their families.”

That enthusiasm highlights the fact that it’s been really tough to find good treatments for Alzheimer’s. Why is that the case? “That's a difficult question. There are a lot of different theories as to why that could be. One theory is that we're too late. By the time we start using these drugs on patients, the disease is already too progressed for it to make a difference,” Milano said.

“Another theory is that we've been going after the wrong targets, so that's why we haven't been successful. Then there's a theory that it will be more like HIV, where it requires multiple different types of drugs at the same time.”

For now, he said Aduhelm gives patients and their families hope. “It's exciting news because it's the first time in a long time we've had an approval. We know the drug pretty well, but it’s not going to be something we prescribe to everyone. I think we're going to have to look closely at the data and talk to patients to figure out who will be the right group of patients to get this medication.”

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Helen Adams