"We are living in historic times for Alzheimer’s. The most exciting report of positive results in Alzheimer's disease clinical trials was recently reported, giving those facing Alzheimer's incredible hope for more effective treatments. As new treatments become available, it is crucial to emphasize that people living with this terminal disease and their families are the best decision makers about what their care journey looks like. That may include new treatments that could slow decline by months. And, in consultation with their healthcare providers, what risk they are willing to bear to have that benefit. The topline data reported on Sept. 27th indicate a positive result from the closely-watched clinical trial of lecanemab, an anti-amyloid monoclonal antibody for the treatment of mild cognitive impairment (MCI) due to Alzheimer’s disease and mild Alzheimer’s dementia. We are enthusiastically looking forward to the full study results later this year. On behalf of all people affected by Alzheimer’s disease around the globe, we are energized about the announcement and the future for more effective treatments. (Read our statement here).
What is data telling us?
In the 18-month Phase 3 CLARITY AD clinical trial of lecanemab (BAN2401), Eisai and Biogen announced that lecanemab reduced clinical decline compared with placebo by 27%, which is significant and clinically meaningful to individuals in the earliest stages of Alzheimer’s. Amyloid build-up was reduced in the brain, based on brain imaging. This was a large study, with nearly 1,800 people enrolled. It is notable that 25% of the U.S. participants in this clinical trial are from underrepresented populations. This is a significantly higher percentage than what is typically seen in clinical trials, which ranges from 1-5% of people from historically underrepresented communities.
What’s at stake?
For people in the earliest stages of Alzheimer’s, it is encouraging we may soon have another FDA-approved drug that may change the course of Alzheimer’s disease. These treatments can give people more time at or near their full abilities to participate in daily life, remain independent, and make future health care decisions. This can make the difference between:
Attending social events, celebrating an anniversary, wedding or baby shower… or not
Driving the car safely… or not
Staying productive at work or as a volunteer… or not
Feeling like you're still yourself… or not
Treatments that deliver benefits to people living with Alzheimer’s disease are just as valuable as treatments that extend the lives of those with other terminal diseases. In just a few years, my mother-in-law went from mild cognitive impairment to a diagnosis of moderate dementia. It was heartbreaking. If drugs like these were available then, promising treatments could have changed the picture, changed the timeline, and changed our lives for the better. The most important thing to note — and what we hear from people impacted by this disease around the country — is that slowing down cognitive decline, even modestly, early in the disease is very meaningful and looks different for every person and family. We all wish for better, more impactful Alzheimer’s treatments — and I believe they are coming— but no one should stop or delay or put up unnecessary obstacles to the current ones, while the next ones are being developed.
Which approach will best tackle a complex disease?
There is vigorous and spirited debate about “the amyloid hypothesis,” and whether or not reducing amyloid provides benefits that are noticeable in everyday life. The results of this study— even just the topline — confirm that amyloid is involved in the Alzheimer’s disease process, and that it is and should be a treatment target. Of course, we need others, too. We know that anti-amyloid approaches are not a “silver bullet,” but they are the first wave of treatments that address the underlying biology of Alzheimer’s. These results support the amyloid therapeutic hypothesis and the U.S. FDA accelerated approval of aducanumab on the basis of amyloid reduction. This is encouraging for other amyloid-lowering therapies that will share their findings in the coming months. We look forward to learning more about the results, including the safety and efficacy of lecanemab. This is only the beginning, our beginning. This first wave of positive results and approvals will change the way we treat Alzheimer’s and potentially other dementia. As seen in other terminal diseases with treatment options, we start with a few wins. And soon, perhaps even in this decade, we will see more effective anti-amyloid treatments. At the same time, we are advancing potential treatments that address other biological causes of Alzheimer's, including those that target tau tangles, inflammation, energy metabolism, vascular contributions and so much more. The Alzheimer’s Association has been laying the groundwork for this and funding multiple approaches for more than a decade. Today, the growing Alzheimer’s drug development pipeline is robust with a wide variety of treatment strategies, some of which will work effectively with anti-amyloid drugs in powerful combination therapies.
While lecanemab is not yet in doctor’s offices, progress like this makes this possible in the not too distant future. I have confidence in a future where we can detect the changes and risk factors that drive memory impairment — which may be different in each individual — and treat them in a way that’s specific to that person. This is what researchers and health care professionals call “precision medicine.” We also must redouble efforts and push for the discovery of additional treatment targets and lifestyle interventions. The Alzheimer’s Association is committed to advancing all potential treatment avenues, and exploring methods for integrating diverse approaches into combination therapies. We remain steadfast in our commitment to advancing the science of Alzheimer’s and dementia treatment, continue to diversify the treatment pipeline through innovative grant programs like Part the Cloud, and focus on all scientifically legitimate avenues of research. All individuals, families, and caregivers facing a devastating, fatal disease deserve an opportunity to access FDA-approved treatments. CMS coverage decisions caused irrefutable and irreversible damage by severely limiting access to those who could benefit from treatment early in the disease. Thousands of people missed out on a year of access to a drug that may have slowed their decline. Too much time has passed, and now, those who could’ve seen benefits are no longer eligible for approved treatments. Very likely, their symptoms progressed rapidly and to the point of no return. This is unethical and unacceptable, and cannot happen again. The Alzheimer’s Association will continue to actively work with Congress and government agencies, including CMS, to remove all unnecessary barriers for those with Alzheimer’s and other dementia to current treatment and those in the research pipeline. We will do everything in our power to ensure treatments that have appropriate safety and demonstrated efficacy are accessible and affordable. This is a watershed moment. When this drug is approved — and based on the current science, we anticipate that it will be approved — people who need it MUST be able to get it. This is history in the making. This is the time we will look back at and say, “that was when it all started.” I am proud to be a part of this community, fighting to end Alzheimer’s and all other dementia."
About the author: As chief science officer, Maria C. Carrillo, Ph.D., sets the strategic vision for the Alzheimer’s Association global research program. Under her leadership, the Association is the world’s largest nonprofit funder of Alzheimer’s research.
October 5, 2022 - Article courtesy of the Alzheimer's Association.