It's not like researchers haven't been trying to come up with new chemical compounds to treat Alzheimer's disease over the past two decades. It's just that the disease has proved a difficult puzzle to work out. Six million people in the United States and 30 million people worldwide have Alzheimer's, the most common form of dementia in the elderly, but the last drug approved for the treatment of the disease was given the rubber stamp by the U.S. Food and Drug Administration (FDA) in 2003.
Only five medications have been available to Alzheimer's patients for the past 20 years, but now the drug manufacturer Biogen has created aducanumab, the first Alzheimer's drug that makers claim gets at the root cause of the disease rather than the symptoms. Biogen says aducanumab, whose brand name will be Aduhelm, targets proteins called amyloids that clump to form cell-damaging plaques on the brains of Alzheimer's patients. Many researchers believe that the more amyloid plaque forms on the brain, the harder it is for brain cells to communicate with one another. The inability to communicate eventually leads to the death of the cells, and the worsening of dementia symptoms in patients.
On June 7, 2021, the FDA approved the use of aducanumab for Alzheimer's, through an accelerated approval process. Patients will receive the drug in the form of a monthly intravenous infusion, and, according to Biogen, treatments will cost a whopping $56,000 per year, in addition to the cost of brain imaging and diagnostic testing. But for those who can afford it, it's a small price to pay for the reduction of symptoms like confusion, communication problems and memory loss, assuming it works.
Critics of the decision say there is very little evidence that aducanumab will work, and that the FDA's decision represents a lowering of standards, caving to pressure from Alzheimer's advocacy groups, patients and their families. Furthermore, if the drug proves largely ineffective, it will be difficult to conduct a post-market trial because most patients who can get a drug from their doctor won't want to risk undertreatment with a placebo in a medical study.
The approval of aducanumab has also been one of the most controversial decisions made by the FDA in recent years. There is serious disagreement among doctors, researchers, advocates and patients about whether the results of a 2019 drug trial indicate aducanumab to be more effective than the placebo. Two members of the FDA advisory panel resigned over the decision to approve the drug over the objections of medical experts.
David S. Knopman, a neurologist at the Mayo Clinic, told the Washington Post that he didn't "wish to be part of a sham process" resulting in the drug's approval. He told the FDA in his resignation that "the whole saga of the approval of aducanumab ... made a mockery of the [advisory] committee's consultative process. While I realize that the committee is advisory, the approval of aducanumab appears [to] have been foreordained," according to the Washington Post.
Neurologist Joel S. Perlmutter of Washington University in St. Louis, a member of the FDA's expert panel for nervous system therapies, also quit in protest. Ten of the 11 panelists on the advisory committee, which met in November 2020, found no evidence in the research presented that the drug would slow cognitive decline. The 11th member was "uncertain."
Biogen claims later trials concluded the drug worked much better in higher doses over longer periods. The FDA concluded there was "substantial evidence that aducanumab reduces amyloid beta plaques in the brain," and that it "is reasonably likely to predict important benefits to patients."
Originally Published: Jun 8, 2021