Innovative Drug Protects Blood-Brain Barrier: A Breakthrough in Alzheimer's Treatment

Recent research has uncovered a promising new approach to combating Alzheimer's disease by focusing on maintaining the integrity of the blood-brain barrier (BBB), a vital protective layer that shields the brain from harmful substances. Scientists at Case Western Reserve University, in collaboration with University Hospitals and the Louis Stokes Cleveland VA Medical Center, have identified a novel drug that effectively preserves BBB function and prevents neurodegeneration in mouse models of Alzheimer's disease (AD).

The study, published in the journal Proceedings of the National Academy of Sciences on May 21, 2025, highlights the importance of targeting the BBB—a departure from traditional strategies that primarily focus on neurons. The research reveals that deterioration of the BBB is an early warning sign of neurodegenerative conditions like AD and traumatic brain injury (TBI). The enzyme 15-PGDH, highly enriched in the BBB and found to be elevated in AD, TBI, and aging in both mice and humans, emerged as a key target.

A drug called SW033291, developed at Case Western Reserve University and University Hospitals, was used to inhibit the enzyme 15-PGDH. Originally designed to activate stem cells for tissue repair, this drug was found to also protect the BBB and reduce brain inflammation without altering amyloid levels—the sticky protein commonly associated with AD. This is significant because current treatments largely aim to remove amyloid but often face limited success and potential side effects.

Remarkably, administering SW033291 even a day after brain injury in mice prevented neurodegeneration and cognitive decline, suggesting potential for therapeutic use in brain injuries, neurodegenerative diseases, and aging-related conditions. The research team, led by co-senior researchers Andrew Pieper and Sanford Markowitz, emphasizes the novelty of focusing on the BBB as a therapeutic target, opening new avenues for drug development in neurodegenerative disease management.

This breakthrough highlights the potential for a safe and effective treatment that preserves brain function by protecting the blood-brain barrier, rather than solely targeting amyloid or neuronal damage. Further studies are underway to explore the drug's applicability in human clinical trials.

Source: https://medicalxpress.com/news/2025-05-potential-drug-blood-brain-barrier.html