Vandy scientists allowed to proceed with Alzheimer’s drug clinical trial

Vanderbilt University
Photo: WKRN

NASHVILLE, Tenn. (WKRN) – Vanderbilt University scientists will be allowed to proceed with an new Alzheimer’s drug clinical trial.

They received notification from the U.S. Food and Drug Administration that testing in humans may proceed after more than 10 years of research by scientists at Vanderbilt University and Vanderbilt University Medical Center.

“The movement to the clinical phase of the research is the result of tireless colleagues reaching across disciplines in pursuit of the shared goal of hoping to someday improve the lives of individuals with Alzheimer’s disease and possibly other brain disorders, such as schizophrenia,” Provost and Vice Chancellor for Academic Affairs Susan R. Wente, Ph.D. said. “This work exactly illustrates the critical role that basic science conducted in partnership with a world-class medical center can play in advancing knowledge in an attempt to fight a devastating disease.”

The aim is for the drug to target major pathologies of the disease and selectively activate a key receptor in the brain.

The Vanderbilt researchers believe that the current standard of care for Alzheimer’s disease, cholinesterase inhibitors, has a different mechanism of action.

They are hoping to establish through future clinical testing that the molecule is broadly effective across a number of cognitive and neuropsychiatric disorders, including schizophrenia.

“This is the first instance I am aware of where an academic drug discovery group moved a molecule designed to hopefully treat a chronic brain disorder all the way from early discovery to human trials without there being, at some point along the way, a pharmaceutical partner,” said P. Jeffrey Conn, Ph.D., Lee E. Limbird Professor of Pharmacology in the Vanderbilt University School of Medicine and director of the Vanderbilt Center for Neuroscience Drug Discovery.