Medical doctors in Pittsburgh are excited in regards to the “life-changing” potential of the brand new Alzheimer’s drug

Proponents of nearly 300,000 Pennsylvania residents living with Alzheimer’s disease on Monday celebrated the federal government approval of a controversial new drug that some say will prove “life-changing” to those showing early signs of the memory-damaging disorder could.

Although the debate continues about how well the novel treatment might work, the Food Drug and Administration approved Biogens 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 Greater Pennsylvania Chapter of the Alzheimer’s Association, which has an office on Pittsburgh’s South Side. “It’s crucial. It is not a cure, but it is a major advance in science. And not just the potential effects (of this drug), but it also signals a fresh start for the future of Alzheimer’s treatments that will hopefully be really effective for everyone involved. ”

If it works as intended, the new drug will mark a major milestone – the first of its kind available in the United States that could slow the progression of Alzheimer’s disease or prevent its onset rather than just trying to reduce it gradually and manage worsening symptoms.

“This could mean more time for the individual to actively participate in daily life, become independent and retain the memory longer. It does so in a way that has never been done before when compared to currently approved drugs that really focus on symptoms, ”Jacobs said. “I hope it gives hope to all those struggling with the disease.”

Experts and doctors in the area are preparing to work with Biogen and insurance regulators in hopes of bringing the drug to the general public, though it remains unclear how long it will take to get to market and how much it will be will cost.

“We are very happy about it,” said Dr. Eric Weisman, director of the Allegheny Health Network Neuroscience Institute’s Memory Disorders Clinic, who has more than 30 years of experience with patients with Alzheimer’s disease and other forms of dementia. “We had no idea what the FDA would decide, but we had worked really hard to prepare for it.”

The expectation of a possible approval of the drug and the generally growing demand for Alzheimer’s and dementia-related treatments prompted AHN’s Neuroscience Institute to hire another doctor who will join Weisman’s team in the fall. Patients and their loved ones have called for such advancement as they struggle with the disease after decades of failed treatment attempts that combat its ill effects.

Mixed opinions on the effectiveness of the drug

However, there continues to be controversial debate in the medical community and among independent experts as to how effective the newly approved drug will be.

An advisory committee on drugs for peripheral and central nervous systems voted against the FDA approval.

“There are people who know and understand the drug, who agree that it should be approved, and there are others who are not convinced,” said Dr. Steven DeKosky, associate director of the McKnight Brain Institute at the University of Florida. who previously spent 18 years at the University of Pittsburgh, where he chaired the Department of Neurology and was previously director of Pitt’s Alzheimer’s Disease Research Center. “You all know that there is an immense need and demand from the community for it.

“It’s not exactly a struggle, but there is real disagreement about what, or how well, this drug will work and whether it’s worth it,” said DeKosky, who did not express his personal opinion on the matter as he also chairs the drug monitoring committee , who examines the safety, risks, and benefits of Biogen’s aducanumab studies. “And there are good reasons for people to have feelings on both sides.”

The concerns stem from mixed results from two Phase 3 clinical trials conducted by Biogen at the same time; one of them showed statistically significant results in slowing the decline in Alzheimer’s disease, and the other that didn’t. Researchers discovered potential benefits in a small subgroup with certain genetic markers in the study with questionable results.

“In general, the field wants you to get your primary score and not later look for the people who answered well because they weren’t statistically analyzed that way – that’s where some of the controversy comes from,” DeKosky said.

The controversy is not about safety concerns.

“The FDA won’t approve a drug unless it believes the risk-benefit balance is in favor of benefit,” DeKosky said.

The FDA plans to continue monitoring the drug’s effects and effectiveness once it becomes available to the public through a Phase 4 study or post-marketing study – a move that proponents say is a sensible solution to allaying ongoing concerns .

“We understand that it can work differently for everyone who takes it, and the idea that it is a complement to comprehensive care, especially for people with mild cognitive impairment or early Alzheimer’s disease. It’s not a substitute for grooming, ”said Jacobs. “It is potentially life changing for the people who can benefit from it.”

How much will it cost?

There is another possible obstacle – how much the new drug will cost and how many people will have access to it.

Biogen has not released official cost estimates, but experts believe that aducanumab treatments could cost between $ 10,000 and $ 50,000 a year.

“The pressure will now shift to Medicare to pay for it,” DeKosky said. “There are a lot of moving parts in it and there will be a lot more.”

The Alzheimer’s Association intends to press for the Centers for Medicaid and Medicare Services to give the go-ahead for funding.

“Ultimately, removing barriers to entry is our top priority,” said Jacobs. “This is an important part of what the association wants to do next – making sure that anyone who could benefit can do so.”

The drug’s approval and high demand for it could also impact recruitment for other future clinical trials in Alzheimer’s disease. According to DeKosky, there are about 100 drugs in various stages of development, from Phase 1 clinical trials that test the treatment in humans first, to other drugs in the process.

It is the first such approval in the field in nearly 40 years, and he noted that the options available in the early 2000s “are not particularly good”.

Increasing demand for the treatment of Alzheimer’s disease

Meanwhile, the number of people diagnosed with Alzheimer’s disease continues to rise.

Almost 6 million Americans are already living with Alzheimer’s disease, with a new diagnosis being made about every 65 seconds, making neurodegenerative disease the sixth leading cause of death in the United States

At the national level, “we see a literal doubling of the number 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.

“For every chronic disease, history has shown us that approval of the first drug in a new category like this will invigorate the field, increase investment in treatments and generate greater innovation,” said Jacobs. “This is just the beginning, but without the first there cannot be a second or third or fourth, each of which is better than the previous treatment.”

Natasha Lindstrom is a contributor to Tribune Review. You can contact Natasha at 412-380-8514, nlindstrom@triblive.com, or on Twitter.