A New Hope for Alzheimer’s: Scientists Develop First Pill to Slow Brain Shrinkage
In what could be a revolutionary breakthrough in Alzheimer’s treatment, scientists have developed a daily oral pill that appears to slow brain shrinkage and protect neurons in people at the highest genetic risk of developing the disease.
The pill, known as valiltramiprosate (ALZ-801), has shown significant success in a Phase 3 clinical trial, marking a major step forward in the fight against one of the world’s most devastating neurological conditions.
According to a study published in Drugs Journal (2025) ↗, the pill works by targeting the earliest stages of Alzheimer’s progression — long before irreversible brain damage occurs.
The Science Behind the Pill
The study focused on individuals who carry two copies of the APOE4 gene variant, a genetic marker known to drastically increase the risk of Alzheimer’s disease. This group represents about 15% of all Alzheimer’s cases and typically experiences faster cognitive decline than others.
Unlike existing antibody-based treatments that remove amyloid plaques after they form, valiltramiprosate prevents the formation of toxic amyloid oligomers — the microscopic proteins believed to trigger the disease’s onset.
Dr. Suzanne Abushakra, lead researcher on the trial, explained:
“Our goal was to stop the process before significant neuronal loss begins. The results show a measurable slowing of brain atrophy, which could mean preserved function and independence for patients.”
Over the course of 78 weeks, participants who took the drug exhibited less brain shrinkage and reduced tissue water diffusion, as revealed by MRI scans. These findings suggest that neurons were not just surviving but staying healthier longer.
A Safer and More Convenient Approach
What sets valiltramiprosate apart from other Alzheimer’s treatments is its safety and accessibility. Current FDA-approved drugs, such as Leqembi and Aduhelm, are antibody infusions that require medical supervision and carry risks like brain swelling or microbleeds.
By contrast, ALZ-801 is an oral pill, designed to be taken daily at home. This could make Alzheimer’s management more practical and less invasive, especially for elderly patients or those in early cognitive decline.
Dr. Abushakra noted that no major side effects were observed in the trial, emphasizing the drug’s potential for widespread use if approved.
A Landmark for Genetic Alzheimer’s
This Phase 3 trial marks the first successful large-scale study conducted exclusively on APOE4 homozygotes, a group that has historically had limited treatment options. The promising results suggest that personalized therapies — those based on a person’s genetic profile — may become the future of Alzheimer’s care.
Researchers are already exploring how ALZ-801 might be combined with other therapies, such as immunotherapies or cognitive training, to create an even more powerful effect. If approved, the pill could enter the market within the next few years, offering hope to millions of families affected by the disease.
The Road Ahead
While more studies are needed to confirm long-term benefits, this development represents a major leap forward in understanding how to intervene early in neurodegenerative diseases.
Alzheimer’s remains a leading cause of death worldwide, with more than 55 million people currently living with dementia, according to the World Health Organization ↗. Treatments that slow or stop brain degeneration could transform public health outcomes and extend quality of life for aging populations.
As the world waits for final approval, the excitement surrounding ALZ-801 is undeniable. It’s not just a new drug — it’s a new way of thinking about how we treat, prevent, and live with Alzheimer’s disease.
