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Controversial Alzheimer’s Drug Critically Tested Before FDA Panel

A federal panel will decide on Friday whether to recommend approval of a controversial but potentially promising Alzheimer’s drug, which is believed to be the first to hit the market in nearly two decades.

The drug, aducanumab, would not stop or reverse dementia, but some evidence suggests that it may slow the progression of memory and thinking problems in people with mild or early symptoms of cognitive decline. giving them a little more time before they develop Alzheimer’s disease. It would be the first drug to do so by attacking the basic biology of Alzheimer’s disease.

However, many Alzheimer’s disease experts are skeptical that aducanumab, made by Biogen, has shown strong enough evidence that it can slow cognitive decline. The drug – given as a monthly intravenous infusion – is also said to be expensive, around $ 50,000 per year. And some experts say it would be difficult for doctors to try to properly prescribe and monitor its use, and that approval of such a drug would reduce the likelihood of patients participating in studies of other drugs for the disease. Alzheimer’s disease that might ultimately work better.

The drug’s path through clinical trials has been difficult, with only one of two Phase 3 trials showing positive results – and those results only emerged from an analysis of additional data after the trials ended in March. 2019 by an independent data watchdog because the drug didn’t seem to work.

Several experts, including a neurologist from the Mayo Clinic who was an on-site investigator for an aducanumab trial, said the evidence is too weak for the drug to warrant approval now and another rigorous clinical trial must. be conducted before a decision is made on whether the drug the drug should be made available.

“Perfection may be the enemy of good, but for aducanumab the evidence isn’t even ‘good’,” wrote neurologist Dr David Knopman in a comment to the panel ahead of Friday’s hearing. . Dr Knopman, who sits on the advisory committee but was recused from the hearing because of his work with the aducanumab trials, added: “Contrary to the hope that aducanumab will help patients with the disease Alzheimer’s disease, the evidence shows that it will not bring any improvement, it will harm some of those who are exposed and will consume enormous resources. “

The Expert Panel, a committee of medical experts that advises the Food and Drug Administration, will review the evidence for the efficacy and safety of aducanumab. If it approves the drug on Friday, that wouldn’t guarantee its approval, but the FDA often follows the recommendations of its advisory committees.

Nearly six million people in the United States and about 30 million worldwide have Alzheimer’s disease, a number that is expected to more than double by 2050. If approved, aducanumab could be used as a drug potential for the estimated two million Americans with mild Alzheimer’s disease. cognitive decline related.

Its availability would have striking implications – not only for patients, but also for doctors, researchers and other pharmaceutical companies and for healthcare costs. It would also be a successful drug for Biogen.

Aducanumab is a monoclonal antibody that targets the protein beta-amyloid which clumps in plaques in Alzheimer’s disease. Many other drugs that reduce amyloid buildup have not been shown to relieve symptoms, so if aducanumab is found to be effective, this would support a long-held theory that attacking amyloid may help if it does. is performed early enough in the disease process, when memory and difficulties are still mild.

Documents released by the FDA ahead of the hearing gave the impression that most of the agency’s reviewers were confident that the data from the successful trial was solid and that the safety concerns, which primarily involved some type of swelling of the brain, were manageable.

“The nominee provided substantial evidence of efficacy to support approval,” wrote Kevin Krudys, an FDA clinical analyst in neurology, in a presentation sent to the committee.

But another FDA reviewer expressed concerns in the documents. Tristan Massie, a mathematical statistician with the FDA, wrote that he believed “there was no convincing and substantial evidence of the effect of treatment or of slowing the disease and that further study was needed” .

Other experts said the degree of benefit the trial claims to show is slight, slowing the 18-month decline by half a point on a 3-point cognitive scale.

“I think it doesn’t matter,” said Dr. Michael Greicius, medical director of the Stanford Center for Memory Disorders, adding that he might discourage his patients from taking it. “For people who say, ‘Oh, come on, it’s okay – if that helps a little, why not give it to people?’ my answer is that there is no data to tell me that this drug works against Alzheimer’s disease.

The Alzheimer’s Association, on the other hand, wrote a letter to the committee in favor of approval. He said the FDA should require a post-market study, but should make the drug available while that happens.

“While the data from the trials have led to some uncertainty within the scientific community, this must be weighed against the certainty of what this disease will do to millions of Americans without treatment,” wrote Joanne Pike, director of the association‘s strategy. “The potential to delay the decline would be denied to millions of people, and this time lost for these spouses, partners, mothers, fathers, grandmothers, grandfathers, aunts, uncles, friends and neighbors cannot be reclaimed. In view of these considerations, we urge approval. “

Dr Eric Reiman, executive director of the Banner Alzheimer’s Institute, who was co-director of studies with other anti-amyloid drugs but not aducanumab, said the advisory committee was faced with a “very unusual and important situation. Since the clinical trials were halted before their expected completion in 2021, when the results could have been more definitive.

“They have a Solomonic decision to make, with one study that showed very promising effects and the other study that did not show an effect,” he said. “I think it’s a tough decision because everyone wants to do what’s best for patients and families.”