Aducanumab as a Novel Treatment for Alzheimer's Disease: A Decade of Hope, Controversies, and the Future.

Alzheimer's disease (AD) is the most common type of dementia and is among the leading cause of death in the United States. Its worldwide prevalence is around 50 million and is projected to double by 2050. Deposition of beta-amyloid (also known as amyloid-beta) peptides (beta 40 and 42) in the brain continues to be the most widely accepted disease mechanism. Until recently, only two Food and Drug Administration (FDA)-approved groups of medications, namely, cholinesterase inhibitors and N-methyl-D-aspartate receptor antagonists, were available for symptomatic treatment with limited efficacy. Disease-modifying therapeutics, keenly desired by clinicians and patients alike, have long been elusive until recently. The FDA's Accelerated Approval Program for the approval of a new agent, aducanumab, is being considered a step in this direction by some, but not without controversy. Aducanumab, marketed as Aduhelm by Biogen, has been shown to lower beta-amyloid plaques in the brain. Biogen believes this will lead to improvement in cognition and functioning in patients with AD. However, within a month of this approval, the FDA has called for investigations into interactions between representatives of Biogen and the FDA preceding the approval of Aduhelm. This report provides an overview of the controversy surrounding the FDA's Accelerated Approval Program as it pertains to the approval of Aduhelm, and the potential impact of these issues on researchers, clinicians, patients, and families in the ongoing battle against this devastating, debilitating, and ultimately fatal illness.