Groundbreaking Alzheimer’s Drug Signals Progress, but Doubts Remain
by TIM CRAIG
It’s been nearly five months since the Food and Drug Administration approved the first new Alzheimer’s treatment since 2003, but its potential impact has been mired in controversies about the approval process, who should get the treatment, its efficacy in fighting the disease, and its cost.
However, despite the troubles, there are some who see the new drug as a shift in how to battle a disease that affects 6.2 million Americans age 65 and older, a number expected to grow to more than 12 million by 2040.
The new medicine, called Aduhelm (generic name aducanumab), was granted a historic accelerated approval for Alzheimer’s treatment by the FDA on June 7 of this year. The drug company Biogen, of Cambridge, Massachusetts, makes the drug.
The drug is the first of its kind to attack what many believe is an underlying cause of Alzheimer’s disease. It works by eliminating clumps of a toxic protein thought to destroy neurons in the brain that lead to cognitive decline. The proteins, known as beta-amyloid plaques and tau, were first discovered in 1984 and are common in people with dementia.
Aduhelm is a monoclonal antibody that stimulates the immune system to target and break down these plaques and is specifically designed for people with the earliest stages of Alzheimer’s disease. People with later stages of Alzheimer’s were not part of the research studies used by the FDA.
According to the FDA, researchers evaluated Aduhelm’s efficacy in three separate studies, which showed that patients receiving the treatment had significant dose- and time-dependent reduction of the amyloid plaque, while patients in the control group had no reduction.
Patients are given a one-hour Aduhelm infusion intravenously once a month for an indefinite period of time. The most common side effect was short-term brain-swelling. Other side effects included confusion and disorientation.
The results of the research warranted the “fast track” designation, which, according to the FDA, “seeks to expedite the development and review of drugs that are intended to treat serious conditions where initial evidence showed the potential to address an unmet need.”
As part of the accelerated approval provisions, the FDA is requiring Biogen to conduct a new, randomized clinical trial to verify the drug’s clinical benefit. If that trial fails, the FDA may initiate proceedings to withdraw approval.
The fact that the FDA approved Aduhelm despite objections from its own independent advisers — many of whom resigned as a result — may have signaled trouble for the new treatment.
The approval process, as well as unclear guidelines as to who, specifically, is eligible for the treatments, has caused some major medical centers to remain undecided on whether to use Aduhelm. Cleveland Clinic and Mass General Brigham in Boston say they’ll pass on it for now, according to reports. In addition, one neurology practice has even banned the company’s sales reps from its offices, citing concerns about the drug and its price, which can climb past $50,000 annually.
The high cost of treatment, low numbers of sites administering the treatment, as well as the confusion surrounding the fast-track approval and eligibility guidelines have seemingly cast a cloud over the new drug.
Many mainstream news stories related to the drug are now focused on potential financial losses to Biogen, such as The New York Times’ October 20 article headlined “Sales of Biogen’s Alzheimer’s Drug Fall Short of Expectations.”
However, focusing on the company’s financial impact misses the point of what Aduhelm signals, according to Kristen Clifford, chief program officer for the Alzheimer’s Association.
“Aduhelm is the first approved treatment to attempt to change the underlying course of the disease,” writes Clifford in a post on the Alzheimer’s Association website. “Simply put: The availability of a new drug signals a new day in Alzheimer’s treatment.”
Clifford says there are several other medications that focus on anti-amyloids in the FDA pipeline, and even more to come. In addition, as groups like the Alzheimer’s Association work with experts in the field to develop guiding principles for administration, insurance coverage, and access for people in all communities, there will be more opportunities to better manage and potentially slow the progression of this disease.
“With new and improved drugs under development every day, this is just the beginning,” says Clifford. “These anti-amyloid drugs are not a cure; they are showing us how to slow, modify or change a disease that continues to devastate families and communities around the world … giving families the possibility of more time to maintain their memories and thinking abilities.”