The approval of a disease-modifying treatment for Alzheimer’s disease: impact and consequences for the nuclear medicine community

Abstract

On June 7th, 2021, for the first time since 2003, a novel drug against Alzheimer’s disease (AD), aducanumab (AduhelmTM), has been approved in the USA by the Food and Drug Administration (FDA). This event already has had a major impact, as shown by the storm of commentaries, both in favor and against it, in journals and social media before and after its disclosure. Its impact is linked to a number of factors: AD is a devastating and incurable disease with a dramatic toll on both patients and their families; Aducanumab is the first approved drug that was tested for its ability to influence the course of the disease (disease-modifying drug) and not only to treat symptoms; the FDA decision was taken on the basis of two phase III trials with discordant results on clinical efficacy, which first were interrupted on the basis of a futility analysis, but subsequently revived (for a detailed summary, see [1]); the FDA decision was strongly based on the amyloidclearing capacity of the drug, as shown by amyloid PET imaging. The scientific and clinical community is cleaved in two fields: on the one hand, those who welcome this decision as a step in the right direction, finally offering a therapeutic option to patients and fostering research and development in this direction, and on the other hand, those considering this decision as driven mainly by expectations and lacking solid scientific evidence. This article is part of the Topical Collection on Neurology