By Jonathan D. Grinstein, PhD
Published October 16, 2025

The last several years have marked a time of breakthroughs on the long and rocky road to effective Alzheimer’s disease treatments. After decades of failure, two antibodies designed to target forms of amyloid-β (Aβ) were shown to halt cognitive decline, results warranting FDA approval. However, these new treatments have drawbacks—they are expensive, have complicated infusion schedules, and come with a risk of adverse reactions like brain swelling and bleeding (ARIA), especially prevalent in people with the APOE4 gene variant associated with increased risk of Alzheimer’s.

Earlier this week, a small biotech company called Alzheon, situated in Framingham, Massachusetts, demonstrated data that has the potential to change the way Alzheimer’s is treated, especially for APOE4/4 homozygotes. In a peer-reviewed research article, Alzheon reported results from its Phase III APOLLOE4 trial evaluating the oral drug ALZ-801 (valiltramiprosate) in individuals with early Alzheimer’s disease who are APOE4 homozygotes. The drug slowed cognitive decline, nearly doubled the brain’s functional preservation, and reduced brain shrinkage without causing the blood vessel problems often associated with antibody treatments.