"After more than a decade of basic and translational research, the Warren Center for Neuroscience Drug Discovery (WCNDD) was finally able to disclose how VU319, a unique M1 PAM, was discovered and profiled," said one of the authors of the study, Craig Lindsley, executive director of the WCNDD and University Professor of Pharmacology, Biochemistry and Chemistry at Vanderbilt University, said in a press release.
"Funding from the National Institute of Mental Health allowed the WCNDD to discover and develop VU319. An important philanthropic gift from the William K. Warren Foundation then enabled us to partner with DavosPharma to conduct critical early-stage studies and earn approval from the FDA as an investigational new drug, paving the way for the Alzheimer's Association award to Dr. Paul Newhouse for the phase I trial," he added.
In conditions such as Alzheimer’s disease and schizophrenia, the neurotransmitter acetylcholine, which is responsible for learning and memory, stops working and disables neurons from proper function. VU319 is an M1 allosteric modulator and works by increasing the efficacy of the endogenous neurotransmitter acetylcholine at the M1 receptor.
In the human trial performed at Vanderbilt University, researchers found signs of target engagement at the highest dose tested without the side effects typical of other drugs that work in the same brain region.
John Kuriyan, dean of basic sciences and University Distinguished Professor of Biochemistry and Chemistry at Vanderbilt University, not in volved in the study, said in a press release, "The successful phase I trial of VU319 marks a potentially transformative step in drug development for Alzheimer's, showcasing Vanderbilt's capacity to translate fundamental research into therapeutic discovery that brings the hope of real clinical impact."
Sources: Science Daily, ACS Chemical Neuroscience
