New Pill Shows Promise in Alzheimer’s Treatment

A recent study suggests that an experimental medication may help slow down memory loss and brain shrinkage in certain Alzheimer’s patients. The drug, named ALZ-801 (valiltramiprosate), appeared to work well for patients with early-stage Alzheimer’s who possess two copies of the APOE4 gene, significantly heightening their risk for the disease.

The research, carried out by Alzeon, a biotechnology firm in Massachusetts, involved a phase 3 trial with 325 patients experiencing either mild cognitive impairment (MCI) or mild dementia, all of whom had the APOE4 gene. Participants, aged between 50 and 80, were randomly assigned to take ALZ-801 or a placebo over roughly 18 months.

While ALZ-801 showed some delay in cognitive decline across the groups, the differences weren’t statistically significant. However, for those with early-stage Alzheimer’s and mild cognitive impairment, the medication seemed to cut memory decline by 50% and nearly stop daily cognitive degradation.

Dr. Christopher Weber, senior director of global science initiatives at the Alzheimer’s Association (who wasn’t part of the study), explained, “Individuals diagnosed with MCI often experience declines in cognitive abilities but can still manage most daily activities independently.”

Patients taking the medication also exhibited slower shrinkage in the hippocampus—an area crucial for memory—showing about 18% less atrophy compared to those on the placebo. The study’s findings are published in the medical journal “Drugs.”

Side effects primarily included nausea, vomiting, and decreased appetite. This experimental pill might eventually offer a more accessible at-home option compared to current approved treatments, like recanemab and donanemab, which necessitate intravenous infusion.

One of the researchers commented, “This may become part of a larger array of anti-Alzheimer’s medications.” The existing therapies focus on eliminating amyloid plaques in the brain, which can provoke swelling and bleeding. In contrast, ALZ-801 aims to prevent these plaques from forming initially, which seems to reduce corresponding side effects.

Weber found it encouraging that patients with the APOE-ε4/ε4 gene didn’t experience heightened cerebral hemorrhage or swelling during the trial, hinting at safety in a high-risk group. Dr. Mark Siegel, a senior medical analyst, noted that this drug could be preemptive, making it possible to avoid full plaque formation, thus sidestepping some adverse effects seen with other treatments.

Researchers acknowledged some limitations in the study; most notably, the strongest results were observed only in the early-stage patients. Plus, APOE4 carriers represent only about 15% of Alzheimer’s cases. The duration of the study also calls for longer follow-up to validate the findings.

Weber added that while primary and secondary results weren’t overwhelmingly positive, follow-ups in specific subgroups did yield hopeful indications, including significant hippocampal shrinkage reduction. There’s potential for this experimental drug to complement existing early Alzheimer’s treatments.

Siegel reiterated the drug’s promise, suggesting that it could form part of a multi-faceted approach to combat Alzheimer’s, focusing on beta-amyloid, tau plaques, and neuroinflammation.

The study was funded by Alzheon, Inc., the creator of ALZ-801, and received additional support from a grant from the National Institute on Aging.