Researchers at the Warren Center for Neuroscience Drug Discovery at Vanderbilt University School of Medicine have successfully completed a Phase I clinical trial for VU319, a drug designed to combat memory loss associated with Alzheimer's disease and schizophrenia.

The trial, led by Dr. Paul Newhouse, demonstrated successful target engagement at the highest dose tested, without the typical side effects seen in similar treatments.

Craig Lindsley, executive director of the center, emphasized that the discovery of VU319 as a unique M1 positive allosteric modulator (PAM) followed over a decade of rigorous research.

VU319 marks Vanderbilt's first complete drug discovery project, progressing from basic science research through to human clinical trials, including high-throughput screenings and candidate selection.

The development and clinical testing of VU319 were made possible through funding from the National Institute of Mental Health and a philanthropic contribution from the William K. Warren Foundation.

Provost Cybele Raver noted that this milestone illustrates Vanderbilt's ability to translate research into clinical applications, offering hope for patients suffering from neurodegenerative diseases.

Currently, Alzheimer's disease affects approximately 6.9 million individuals aged 65 and older, with VU319 showing potential not only for Alzheimer's but also for other conditions such as schizophrenia, prion diseases, and vascular dementia.

VU319 works by enhancing the efficacy of acetylcholine, a neurotransmitter essential for learning and memory, at the M1 receptor, thereby optimizing therapeutic benefits.

John Kuriyan, dean of basic sciences, highlighted that the success of VU319 is a significant advancement in drug development for Alzheimer's disease, showcasing Vanderbilt's research capabilities.

Following the Phase I trial, the Warren Center plans to develop additional M1 PAMs for future human clinical testing.

The discovery of VU319 was detailed in an article titled 'Discovery of VU0467319: an M1 Positive Allosteric Modulator Candidate That Advanced into Clinical Trials', published in ACS Chemical Neuroscience.

With no known cure for Alzheimer's, VU319 represents a promising step forward in addressing the needs of millions affected by various neurodegenerative conditions.