Someone in the U.S. is diagnosed with Alzheimer’s disease every 65 seconds; in 2020, it was estimated that 5.8 million Americans aged 65 or older had the condition. These startling statistics are made all the more unsettling by the fact that there is currently no known cure, and few effective treatments, for the disease. But there might be good news on that front: for the first time in nearly 20 years, a new treatment for Alzheimer’s disease has been approved for use in the United States by the U.S. Food and Drug Administration (FDA). Aducanumab, known by the brand name Aduhelm, targets dementia, the underlying cause of Alzheimer’s. But while U.S. manufacturer Biogen, Inc, creator of the drug, has found that the drug works well in higher doses, use of the drug has been narrowed after the FDA received criticism for approving it.
Aducanumab has had a somewhat rocky approval process. In March 2019, late-stage trials involving 3,000 patients were halted when it was determined that the drug was not slowing down deterioration in participants. However, after Biogen and the FDA worked together to further analyze the data, they concluded that the drug might actually work; Biogen then began collaborating on the drug with the FDA, who fast-tracked its approval this year. They agreed that the drug targets and reduces amyloid, a protein that forms abnormal clumps in the brain of people with Alzheimer’s, which damages brain cells.
The FDA said there was “substantial evidence that aducanumab reduces amyloid beta plaques in the brain” and that this “is reasonably likely to predict important benefits to patients.”
The drug would cost roughly $56,000 a year and requires monthly IV infusions.
The FDA’s approval of the drug has been criticized by many, including members of the FDA’s advisory panel, some of whom resigned in protest, and other drug manufacturers are also voicing their concerns.
Experts are split on whether the FDA’s fast-tracking of the Alzheimer’s drug is potentially harmful or beneficial. Some think more evidence is necessary to prove that the drug is effective, and others worry that this might mean the FDA will begin to fast-track treatments for other diseases, which could put false hope above solid clinical science, and possibly harm patients. On the other hand, some advocacy groups see this as a possible way to help patients dealing with other diseases: “If the FDA can find a way to be flexible for Alzheimer’s, maybe they can find a way to be flexible for ALS,” said Neil Thakur, chief mission officer at the ALS Association.The acting head of the FDA, Dr. Janet Woodcock, has weighed in on the controversy, calling for a government investigation into the contacts between Biogen and some of her agency’s drug reviewers. “We believe an independent assessment is the best manner in which to determine whether any interactions that occurred between the manufacturer and the agency’s review staff were inconsistent with FDA’s policies and procedures,” Woodcock said on Twitter.
After the criticism of the drug’s approval, Biogen has narrowed the use of Aduhelm, recommending the treatment be used for people with mild cognitive impairments. They have asked for an update to the label, stating there is no safety or effectiveness data on treatment at earlier or later stages of Alzheimer’s.
As for now, the debate over the drug’s approval continues: aducanumab is being hailed by patient advocates who are eager for a treatment for Alzheimer’s, while others are saying that clinical trials of the drug are inconsistent and more research is needed. Biogen has now been required to conduct a follow-up study to provide more solid evidence that the drug is actually effective. At the very least, this controversial drug could help boost underfunded dementia research, which would be a win for everyone.