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Pittsburgh doctors excited about 'life-changing' potential of new Alzheimer's disease drug

Pittsburgh Tribune-Review - 6/8/2021

Jun. 8—Advocates for nearly 300,000 Pennsylvanians living with Alzheimer's disease celebrated the federal government's approval Monday of a controversial new drug some say could prove "life-changing" for those showing early signs of the memory-destroying disorder.

Though debate persists over just how well the novel treatment could work, the Food Drug and Administration approved Biogen's aducanumab, which aims to prevent or slow the progression of Alzheimer's disease and its cognitive effects by targeting harmful clumps of a protein called beta-amyloid in the brain.

"It's a new day for millions of Americans," said Clay Jacobs, executive director of the Alzheimer's Association Greater Pennsylvania Chapter, which has an office in Pittsburgh'sSouth Side. "It's pivotal. It's not a cure, but it's a significant advancement in the science. And not only the potential impact of (this drug), but it also signals what I would consider a new beginning for the future of Alzheimer's treatments in a way that's hopefully really impactful for all of those dealing with this."

If it works as intended, the new drug will mark a major milestone — the first such treatment made available in the United States that could slow the progression of or prevent the onset of Alzheimer's disease, as opposed to attempting merely to lessen and manage its progressively worsening symptoms.

"That could mean more time for individuals to actively participate in daily life, to have independence, to sustain memory longer. It does this in a way that has never been done before compared to the currently approved drugs that really focus on the symptoms," Jacobs said. "I hope it gives hope to all of those who have struggled with the disease."

Experts and physicians in the region are preparing to work with Biogen and insurance regulators in hopes of getting the drug available to the general public, though it remains unclear just how long it'll take to get to market, and how much it'll cost.

"We're excited about this," said Dr. Eric Weisman, director of the Memory Disorders Clinic for Allegheny Health Network Neuroscience Institute, who has more than 30 years' experience with patients who have Alzheimer's disease and other forms of dementia. "We had no idea what the FDA was going to decide, but we had been really hard at work in preparation for this."

Anticipation of the drug's possible approval as well as growing demand in general for Alzheimer's and dementia-related care spurred AHN's Neuroscience Institute to hire an additional physician who will be joining Weisman's team this fall. Patients and their loved ones have been clamoring for such an advancement as they struggle with the disease, after decades of failed attempts at treatments that combat its negative effects.

Mixed opinions on drug's efficacy

Controversy persists, however, in the medical community and independent experts over just how effective the newly approved drug will be.

A peripheral and central nervous systems drug advisory committee voted against FDA approval.

"You have people who are in the know and understand the drug who are in agreement that it should be approved, and you have others who are not convinced," said Dr. Steven DeKosky, deputy director at the University of Florida'sMcKnight Brain Institute, who previously spent 18 years at the University of Pittsburgh, where he chaired the neurology department and formerly served as director of Pitt's Alzheimer's Disease Research Center. "All of them know there is an immense need for it and immense demand for it from the community.

"It's not exactly a battle, but there is a real difference of opinion about what or how well this drug will do and whether it was worthy," said DeKosky, who did not speak to his personal opinion on the issue as he also chairs the drug monitoring committee that examines the safety, risks and benefits of Biogen's aducanumab trials. "And there are good reasons for people to have feelings on both sides."

The concerns stem from differing results from two Phase 3 clinical trials conducted by Biogen simultaneously; one of which showed statistically significant results in slowing the decline of Alzheimer's disease, and the other, which did not. Researchers did discover potential benefits among a small subset with certain genetic markers in the trial with questionable results.

"Generally, the field wants you to hit your primary outcome, and not go back later and look for the people who responded well because they weren't statistically set up to be analyzed that way — there's where some of the controversy comes from," DeKosky said.

The controversy is not over safety concerns.

"The FDA will not approve a medication unless they believe that the risk benefit ratio is in favor of benefit," DeKosky said.

The FDA plans to continue to monitor the drug's effects and effectiveness as it becomes available to the public via a Phase 4 trial, or post-marketing study — a move that advocates say marks a reasonable solution to addressing persisting concerns.

"We recognize that it may work differently for everyone that takes it, and the idea that it's a complement to comprehensive care, specifically, for those with mild cognitive impairment or early Alzheimer's. It's not a substitute for care," Jacobs said. "For the people it can benefit, it is potentially life-changing."

How much will it cost?

There's another possible obstacle at play — just how much the new drug will cost and how many people will be able to access it.

Biogen has not released official cost estimated, but experts suggest that aducanumab treatments could ring up between $10,000 and $50,000 a year.

"The pressure now will move to Medicare to pay for it," DeKosky said. "There are a lot of moving parts on this and there's going to be a lot more."

The Alzheimer's Association intends to push for the Centers for Medicaid and Medicare Services to green-light funding.

"Ultimately, eliminating barriers to access is our highest priority," Jacobs said. "That is an important part of what the association looks to take on next — making sure anyone who could benefit is able to."

The drug's approval and high demand for it also could affect the ability to recruit participants to other future clinical trials in the Alzheimer's disease field. There are about 100 drugs in various states of development, from Phase 1 clinical trials first testing the treatment on humans to those further along in the process, according to DeKosky.

It's the first such approval he's witnessed in nearly 40 years in the field, and he noted the options available in the early 2000s "are not terribly good."

Demand climbing for Alzheimer's disease care

Meanwhile, the sheer number of individuals being diagnosed with Alzheimer's disease continues to climb.

Nearly 6 million Americans already are living with Alzheimer's disease, with a new diagnosis made about every 65 seconds, making the neurodegenerative disorder the sixth leading cause of death in the U.S.

Nationally, "we're looking at literally a doubling of people with Alzheimer's disease by 2050," DeKosky said.

In Pennsylvania, an estimated 320,000 people will be living with the disease by 2025, according to the Alzheimer's Association.

"With any chronic disease, history has shown us that approvals for the first drug in a new category like this will invigorate the field, increase investments in treatments, and generate greater innovation," Jacobs said. "This is just the start, but without the first, there can't be a second or a third or a fourth, each improving on the earlier treatments."

Natasha Lindstrom is a Tribune-Review staff writer. You can contact Natasha at 412-380-8514, nlindstrom@triblive.com or via Twitter .

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