MuhammadSalis 
A potential breakthrough in Alzheimer’s treatment is drawing renewed attention from scientists after surprising results revealed that a simple pill may protect certain high-risk patients from memory decline.
A Second Chance for a Struggling Drug
Earlier this year, the outlook for the experimental Alzheimer’s drug valiltramiprosate, known as ALZ-801, seemed grim. Initial topline data from a study involving more than 300 participants—aged 50 and older and genetically predisposed to Alzheimer’s—showed little difference between those taking the drug and those given a placebo.
However, a deeper analysis released in September told a different story. Among a subgroup of 125 participants with only mild cognitive impairment (MCI) at the start of treatment, the results were far more promising.
“These individuals showed very meaningful responses,” said Dr. Susan Abushakra, Chief Medical Officer of Alzheon, the company behind ALZ-801.
For this subgroup, cognitive decline slowed by about 52%, a result comparable to the benefits reported for the FDA-approved Alzheimer’s drugs lecanemab and donanemab.
Encouraging Brain Imaging Results
While the smaller sample size limits statistical certainty, other findings add weight to the drug’s potential. Brain scans revealed that participants taking ALZ-801 experienced approximately 18% less atrophy in the hippocampus—the brain region essential for memory—compared to those on placebo.
“That’s an important difference,” said Abushakra. “Preserving hippocampal volume is critical to maintaining memory and cognitive function.”
The findings, recently published in the journal Drugs, came from a clinical trial supported by a $47 million grant from the National Institutes of Health (NIH).
A Pill Instead of an Infusion
One of ALZ-801’s major advantages is how it’s delivered. Unlike current Alzheimer’s treatments, which are monoclonal antibodies administered via intravenous infusions, ALZ-801 is an oral pill taken twice daily at home.
This approach offers a simpler, less invasive, and potentially less expensive option. It also sidesteps one of the biggest complications seen with antibody-based treatments—brain swelling and bleeding—a risk particularly high among some Alzheimer’s patients.
While monoclonal antibodies work by breaking down existing amyloid plaques, ALZ-801 takes a different route: it prevents the formation of plaques altogether by stopping amyloid proteins from clumping in the first place.
This mechanism not only avoids the dangerous side effects associated with antibodies but could also help stop Alzheimer’s earlier in its course.
Why It Matters for APOE4 Carriers
ALZ-801 could be especially important for people who carry two copies of the APOE4 gene, a group that faces 10 times the average risk of developing Alzheimer’s. Although these individuals represent only 2% of the population, they account for about 15% of all Alzheimer’s cases.
Unfortunately, this same genetic profile also makes them more vulnerable to side effects from antibody treatments, including inflammation and brain swelling.
“These individuals are at higher risk for inflammation in the brain that can be quite serious,” explained Jessica Langbaum, an Alzheimer’s researcher at Banner Health in Phoenix.
Langbaum noted that with cautious dosing, monoclonal antibodies can still be used safely. But others argue that a safer, oral alternative like ALZ-801 could be a better long-term option.
A Scientist and Patient’s Perspective
For David Watson, a researcher who also carries two APOE4 genes, the search for safer therapies is personal.
“People like me need a treatment that doesn’t carry the same risk of brain inflammation,” Watson said.
Watson, a co-author of the study, believes ALZ-801 could offer more than just safety—it might actually do a better job at preserving neurons.
“The drug appears better at reducing levels of a protein fragment associated with brain cell death,” he said. “We’re really making a difference in keeping neurons alive.”
Promising Signs in Long-Term Use
Watson added that patients who continued taking ALZ-801 after the 18-month trial have shown encouraging stability.
“Despite carrying genes that usually lead to a swift decline, many of them are holding their own,” he said. “They’re in their 60s and 70s, and their cognition remains steady.”
This long-term resilience hints that ALZ-801 might not just slow memory loss—it could preserve brain health over time, offering new hope for patients at the highest genetic risk.
What’s Next for ALZ-801?
While these results are still preliminary, they’ve reignited optimism about oral treatments for Alzheimer’s disease.
The FDA typically requires larger, confirmatory trials before granting approval, but ALZ-801’s unique advantages—convenience, safety, and its potential for early intervention—may prompt regulators to give it special consideration.
If future studies confirm these findings, ALZ-801 could represent a major step forward in making Alzheimer’s treatment simpler, safer, and more accessible, particularly for those most vulnerable to the disease.
Key Takeaways
- ALZ-801 slowed cognitive decline by 52% in patients with mild cognitive impairment.
- Participants showed 18% less hippocampal atrophy than those on placebo.
- The drug is an oral pill, unlike infusion-based antibody treatments.
- It prevents amyloid plaque formation instead of breaking existing ones.
- APOE4 carriers, at 10x higher Alzheimer’s risk, may benefit most.
- It appears safer, avoiding swelling or bleeding in the brain.
- Early data suggests continued benefits beyond 18 months.
Final Thoughts
While it’s too early to declare ALZ-801 a game-changer, it’s a promising reminder that innovation in Alzheimer’s research is far from over. If larger trials confirm these results, the future of Alzheimer’s care could shift from costly infusions in clinics to a pill taken at home—offering hope, safety, and dignity to millions at risk.
