Potential Use of Diabetes Medication as a Non-Invasive Treatment for Hydrocephalus

Recent research from Northwestern University suggests that a drug traditionally prescribed for type 2 diabetes could offer a new, less invasive approach to treating hydrocephalus, a condition characterized by the accumulation of excess cerebrospinal fluid (CSF) within the brain. The study, published in the Journal of Clinical Investigation, highlights how sodium-glucose cotransporter 2 (SGLT2) inhibitors may influence the dynamics of CSF in patients with hydrocephalus.

Hydrocephalus, affecting up to 3% of individuals over 65, results from the buildup of cerebrospinal fluid, leading to increased pressure in the skull, which can impair brain functions. The standard treatment involves surgical implantation of ventriculoperitoneal shunts, which divert excess fluid away from the brain into the abdomen for absorption. While effective, this surgical intervention carries risks and is often permanent.

Interestingly, some patients with normal pressure hydrocephalus also have type 2 diabetes and are on SGLT2 inhibitors to manage their blood sugar, cardiovascular health, kidney function, and weight. Observations of brain ventricle size reduction in such patients prompted researchers to explore whether these drugs could offer therapeutic benefits beyond their current uses.

The senior author of the study, Dr. Stephen Magill, explained that SGLT2 inhibitors block a receptor found in the kidneys, but this receptor is also expressed in the choroid plexus—the brain structure responsible for producing CSF. Animal studies had previously indicated this connection, and now clinical evidence suggests these medications may influence ventricular size.

To investigate, researchers performed CT scans on three hydrocephalus patients before and after they underwent surgery to implant ventriculoperitoneal shunts, followed by the initiation of SGLT2 inhibitor therapy. All three patients displayed notable reductions in ventricular size, with one experiencing a significant collapse of brain ventricles, which required a shunt valve adjustment.

These findings raise the possibility that SGLT2 inhibitors could be used to treat normal pressure hydrocephalus without surgery in the future. Dr. Magill emphasized that this discovery opens new avenues for research into how these drugs may prevent or manage hydrocephalus, potentially reducing the need for invasive procedures. The team is now studying SGLT2 knockout mouse models to further understand the mechanisms involved.

This innovative research could lead to the development of new therapeutic strategies for hydrocephalus, including post-traumatic cases, and enhance our understanding of CSF production and secretion pathways, ultimately improving patient outcomes with fewer surgical risks. More studies are needed, but this represents an exciting step forward in neuro-therapeutics.

Source: https://medicalxpress.com/news/2025-06-diabetes-drug-alternative-treatment-option.html