Experimental Pill Shows Promise in Slowing Alzheimer’s Progression

An experimental pill could offer new hope in the battle against early-stage Alzheimer’s disease, according to promising research results from a recent clinical trial. The medication, known as ALZ-801 (valiltramiprosate), has demonstrated potential to slow memory decline and reduce brain shrinkage, particularly in patients with specific genetic markers.

Massachusetts biotech company Alzheon conducted a phase 3 trial involving 325 people aged 50 to 80 with early Alzheimer’s disease, all of whom carried the APOE4 gene—a genetic variant that increases Alzheimer’s risk tenfold. Participants received either ALZ-801 pills or a placebo over an 18-month period.

While results across all participants showed modest improvements that weren’t statistically significant, a subset of patients with the earliest stages of Alzheimer’s—those with mild cognitive impairment (MCI)—demonstrated remarkable benefits. In this group, the pill appeared to slow memory decline by 50% and almost completely halt daily cognitive decline.

“Individuals diagnosed with MCI experience a decline in cognitive abilities, including memory, language or visual/spatial perception—however, they maintain the ability to independently perform most activities of daily living,” explained Christopher Weber, Ph.D., senior director of global science initiatives at the Alzheimer’s Association in Chicago, who was not involved in the study.

The medication also showed promising effects on brain structure. Participants taking the twice-daily pill experienced slower brain shrinkage, with approximately 18% less atrophy in the hippocampus—the brain region critical for memory and thinking—compared to those receiving the placebo.

These findings, published in the medical journal Drugs, highlight a potential alternative to current Alzheimer’s treatments. Unlike existing approved medications such as lecanemab or donanemab, which require intravenous infusions, ALZ-801 could be taken at home in pill form.

Another significant advantage appears to be the safety profile. Current monoclonal antibody treatments work by breaking down amyloid plaques already formed in the brain and have been linked to brain swelling and bleeding. ALZ-801 takes a different approach by preventing amyloid plaques from forming initially.

“In this trial, the fact that APOE-ε4/ε4 individuals did not show increased brain bleeding or swelling is encouraging, and suggests that this drug may be relatively safe in a population that is otherwise at higher risk of side effects,” Weber noted.

Fox News senior medical analyst Dr. Marc Siegel emphasized this benefit: “This drug can be given before the plaques fully form, so that prevention may be a goal. As a result, we don’t see the brain swelling that is sometimes a side effect of the plaque-dissolving monoclonal antibodies.”

The study does have important limitations. The strongest results were only observed in the earliest-stage patient group rather than across all participants. Additionally, the trial focused exclusively on carriers of the APOE4 gene, which represents approximately 15% of all Alzheimer’s patients. Experts also note that the 18-month trial period is relatively short, with longer follow-up studies needed to confirm the durability of these effects.

Weber acknowledged that while the primary and secondary outcomes of the trial were technically negative, “follow-up analyses with specific subgroups were encouraging, including that the treatment did cause significant slowing of shrinkage in the hippocampus.”

The most common side effects reported included nausea, vomiting, and appetite suppression.

Looking forward, researchers see potential for ALZ-801 to complement other Alzheimer’s treatments. “This treatment could potentially be used in combination with other anti-amyloid treatments, though more evidence is needed to understand how this drug could be used as a part of a combination therapy,” Weber said.

Dr. Siegel expressed optimism about the medication’s future: “It may well have a future as part of the growing arsenal of anti-Alzheimer’s drugs, which look at several different targets of prevention, including beta amyloid and tau plaques as well as neuro-inflammation.”

The study was funded by Alzheon, Inc., the developer of ALZ-801, with additional support from a U.S. National Institute on Aging grant. While these initial results are promising, larger clinical trials will be necessary to fully validate the findings before the drug could receive regulatory approval and become available to patients.