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Wednesday, 23 June 2021

Is new Alzheimer’s drug a groundbreaking treatment, or the FDA’s biggest mistake?

When the first new Alzheimer’s drug in 20 years was approved by the FDA earlier this month, many expected the celebration of this small victory against a devastating disease. Instead, a string of resignations followed from members of the committee who had reviewed and evaluated the safety and effectiveness of the drug. They protested that the new medicine — aducanumab, known by the shelf name Aduhelm — was both unsafe, and had shown no real efficacy against the cognitive dementia caused by the disease.

So what exactly is this new drug? Researchers believe that aducanumab attacks the amyloid-protein plaques that impair the cognitive function of the brain. High doses of the drug seemed to be effective at slowing cognitive decline for early-stage patients, though further study was recommended.

Did the trials at least have promising results? Absolutely not. During the clinical trials, about 42% of patients experienced brain swelling or bleeding after the first dose. The prohibitive cost of the drug, a whopping USD 56k a year, has also sparked outrage.

What do the experts say? The Peripheral and Central Nervous System Drugs Advisory Committee had advised against approving the drug after finding no sufficient evidence that it could actually impede the cognitive decline of dementia, according to The New Yorker. Ten committee members voted no, one voted uncertain, while another abstained. With not a single vote in favor of the medicine from a committee of experts, why did the FDA approve it anyways?

The prevalence of the disease may have something to do with it: Alzheimer's is ranked as the sixth leading cause of death in the US, and is the primary cause of dementia, which affects an estimated 50 mn people worldwide, according to World Health Organization estimates. The quest for an Alzheimer’s treatment has been high on medical researchers’ agendas for decades, with the first treatment for the disease trialled in 1987 and bns of dollars pumped into global research since. The global societal cost of dementia in 2015 was estimated at USD 818 bn, equivalent to 1.1% of global GDP.

Desperate for a solution, the FDA seems to have ignored conventional wisdom: Aducanumab was approved using the authority’s “Accelerated Approval Pathway,” which OKs drugs that fill unmet medical needs based on indications — not guarantees — that they are likely to have clinical benefits.

The FDA’s perspective: In a statement, the FDA said it anticipated “an expectation of clinical benefit,” from the drug’s approval, and that it believed that “the benefits [of aducanumab] for patients with Alzheimer's disease outweighed the risks of the therapy.” The FDA also said it would require the manufacturer Biogen to conduct further clinical trials to verify the drug’s safety and efficacy, and could remove it from the market if it proves ineffective.

Besides, some say they have found the medicine helpful: A 68-year-old patient who was part of the trials said that the drug helped him feel less confused, with his family also noticing improvement, according to the BBC. Meanwhile, the first Alzheimer’s patient outside a trial context received the first dose of the intravenous drug last week.

Our take: It’s likely too early to tell, but the FDA seems to have jumped the gun on this one, potentially putting the public at risk. Still, no treatment comes without its risks and side-effects, and the possibility that this medicine may make life a little more bearable for Alzheimer’s sufferers and their loved ones likely informed the FDA’s decision.

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