Experimental Treatment May Slow Fatal Brain Disease in Women

Researchers at Tel Aviv University have identified promising evidence that the experimental drug Davunetide may significantly slow the progression of progressive supranuclear palsy (PSP) in women. The findings, led by Prof. Illana Gozes of the Sagol School of Neuroscience and the Gray Faculty of Medical and Health Sciences, underscore the importance of incorporating biological sex into the development and evaluation of treatments for neurodegenerative diseases. The study reanalyzed data from a 52-week international clinical trial involving more than 300 patients with PSP, a rare and fatal neurodegenerative disorder caused by the abnormal accumulation of tau protein in the brain. Using updated outcome measures now recommended by the U.S. Food and Drug Administration (FDA), the researchers reached conclusions that differed substantially from those of the original clinical trial, which had found the drug to be safe but ineffective.

The research team included Dr. Guy Shapira, Jason Blatt, Liri Guz, and Prof. Noam Shomron. The findings were published in Molecular Psychiatry, a Nature Portfolio journal. The reanalysis revealed that women treated with Davunetide experienced a significant slowing of disease progression, while no comparable effect was observed in men. Women receiving the treatment maintained key motor and functional abilities, including balance, fine motor skills, and everyday activities such as using cutlery, buttoning clothes, and washing their face and hands. They also demonstrated significant improvements in cognitive measures, including language, working memory, and overall cognitive performance. The researchers also identified profound molecular differences between women and men. They found that the relationship between levels of pathological tau protein in the cerebrospinal fluid—a key biomarker of the disease—and clinical symptoms differed markedly between the sexes. In women, higher levels of pathological tau were associated with a significant decline in language abilities, whereas no such relationship was observed in men. These findings suggest that the underlying disease mechanisms may differ between women and men and may help explain their different responses to treatment. "Our data show that analyzing women and men separately is not merely a statistical exercise, but an essential tool for developing more effective treatments for neurodegenerative brain diseases," said Prof. Gozes.

The researchers believe their findings provide a strong scientific foundation for future clinical trials designed to evaluate Davunetide as a targeted treatment for women with PSP. More broadly, the study supports the development of precision medicine approaches for tau-related disorders, including Alzheimer's disease and other neurodegenerative conditions. The study was supported by ExoNavis Therapeutics, which is developing Davunetide for the treatment of brain diseases under a license from Ramot, Tel Aviv University's technology transfer company. Prof. Gozes serves as Vice President for Drug Development at ExoNavis Therapeutics, and Dr. Guy Shapira serves as a consulting statistician. The study's findings were independently validated by several additional statisticians.

Read the full study ►  https://www.nature.com/articles/s41380-026-03709-x