Lessons Learned About Clinically Meaningful Benefit from Antiamyloid Trials: Ronald C. Petersen, MD, PhD


The director of the Alzheimer’s Disease Research Center at Mayo Clinic provided perspective on the emergence in antiamyloid therapies, assessing clinically meaningful benefit, and potential changes to trial design in the future. [WATCH TIME: 3 minutes]

WATCH TIME: 3 minutes

"Taking a look at the bigger picture of all these possible proteins operating in concert, if we impact 1 of them, either amyloid or any of the others in isolation, how much of a clinical impact are we likely to have? We’re seeing in trials now that the degree of impact is clinically meaningful, it’s important, but it’s certainly not the whole picture. We need to look at the broader context of these proteins and how they may be interacting together."

Due to complex pathology of Alzheimer disease (AD), trying to understand the true impact of a therapy has been a challenge that many in the field have been tirelessly working to solve. Today, there are hundreds of ongoing trials assessing agents for the disease, all in different stages of development, with the hopes of building out a toolbox of disease-modifying options. After several years of failed approaches and studies, the field finally broke through in 2021 with the approval of aducanumab (Aduhelm; Biogen); however, several continued to question whether these were truly meaningful and beneficial findings.

Questions remain about the time therapies need to show a clinically meaningful effect, as well as whether patients should be treated before symptoms arise. The expansion of biomarkers has allowed for more free discussion about the possibilities of new trial design and inclusion of patients earlier in their disease course; however, there are still issues with access and costs with some of these techniques. Recently, the Alzheimer’s Association formed a group of experts to identify cognitive and functional measures widely used in drug development, and the true meaning of clinically beneficial of meaningful slowing of the progression of AD.

In an interview with NeurologyLive®, lead investigator Ronald C. Petersen, MD, PhD, director of the Alzheimer’s Disease Research Center at Mayo Clinic, provided perspective on the lessons learned from the antiamyloid trials and their impact on the definition of clinically meaningful benefit. Additionally, he commented on the complex nature of the disease, and how it will not be managed with just 1 approved therapy.

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