A patient in clinic who, when asked his age, exclaimed, “Doc, I’m 84 but feel like 50 and am enjoying life!” And indeed, he did appear to be the picture of health. Sadly, though, he had a disease — Alzheimer’s disease — that would progressively reduce his memories and his independence.
Unfortunately, he is more the rule than the exception for all of us as we grow old. Among those over the age of 80, 30-50% harbor amyloid buildup in the brain, a hallmark of Alzheimer’s disease, which translates into 120,000 people in our state. And while we have had medications for the past 20 years that provide some degree of symptomatic benefit, Alzheimer’s disease has proven very resistant to attempts to slow its course … until now.
Last week we saw what many think of as the beginning of a new era in treatment of Alzheimer’s disease; the first therapy that may actually retard progression of the disease received accelerated approval for use by the FDA. However, the approval of this medication, aducanumab, was not without controversy.
We have often heard during the COVID pandemic medical experts advising us to “follow the science.” In the case of aducanumab, one is left with deciding what science to follow. Proponents of this therapy point to evidence that aducanumab does indeed remove amyloid from the brain and to two major studies indicating that individuals with mild Alzheimer’s disease receiving the target dose of aducanumab for a period of time had 25% less decline in tests of cognition and 40% less decline in functional measures compared to those who were not given the medication. Skeptics of the drug argue that the statistical analyses done to demonstrate these benefits are compromised by critical miscalculations that occurred during the study and that taint the results.
In the face of these opposing opinions, and against the advice given to them by their own External Advisory Committee, the FDA went ahead with a bold decision to make aducanumab available to individuals with Alzheimer’s disease now while requiring the manufacturer, Biogen, to conduct a further study to confirm their results. But was this the right decision?
For medical providers like us and other colleagues on the front line of treating people with Alzheimer’s disease, we would say yes. There are two fundamental principles of medical ethics to which we can turn, beneficience and autonomy. Beneficience refers to an action that has the intent of doing good for our patients. With every day that passes, people with Alzheimer’s disease are slowly losing more and more memory and independence. While the data from the aducanumab studies may not be ideal, to withhold a potentially beneficial treatment that has reasonable evidence of effectiveness for several years while waiting for another study to be completed does not seem in the best interest of our patients. Particularly for people with mild symptoms, reducing the rate of disease progression could improve quality of life for years.
Autonomy means that a person can make his or her own decisions about what to do and what to agree to. Aducanumab is not for everyone, and not everyone with mild Alzheimer’s disease will choose to take it. As is the case in other fields of medicine, the decision to use this medication will involve an informed discussion between medical providers and patients and their loved ones regarding the benefits and risks.
The UW Medicine Memory and Brain Wellness Center envisions a world in which people live well with memory loss, and can rely on the best care, within a community of support. The fundamental significance of the release of aducanumab is to renew hope for effectively changing the biological course of Alzheimer’s disease, and to draw public attention to the key importance of an early and accurate diagnosis. Aducanumab has a place in this vision, but is just one step. Meanwhile we continue to promote the development of new therapies for Alzheimer’s disease and related dementias, and it has been rewarding to us to participate in studies of many promising new medications, including strategies to eventually prevent the disease. We look forward to bringing more of these opportunities to the Puget Sound community.