Patients with serious brain disorders such as Alzheimer's disease and schizophrenia could benefit from an investigational new drug (IND) that has received notification from the FDA that testing in humans may proceed after more than 10 years of research by scientists at Vanderbilt University and Vanderbilt University Medical Center.

"This is the first instance I am aware of where an academic drug discovery group moved a molecule for treatment of chronic brain disorders 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, PhD, director, Vanderbilt Center for Neuroscience Drug Discovery (VCNDD). "And that really is crossing what people refer to all of the time as the 'Valley of Death,' where good research discoveries have a hard time moving into the real testing phase."

For Alzheimer's disease, the IND targets major pathologies of the disease and offers highly selective activation of a key receptor unlike the current standard of care, cholinesterase inhibitors. Vanderbilt researchers said the molecule might be broadly effective across a number of cognitive and neuropsychiatric disorders. In schizophrenia, current treatments address the positive symptoms of the disease, including hallucinations and delusions, but not the negative symptoms and cognitive disturbances of schizophrenia. "People who treat schizophrenia patients commonly agree that the negative symptoms and cognitive disturbances are the major factors that decrease the ability for these patients to integrate in society," Conn said. "And we have no treatments for those so our hope is that this molecule will be effective."

VCNDD Co-Director Craig W. Lindsley, PhD, director of Medicinal Chemistry and the William K. Warren, Jr. Chair in Medicine, said phase I testing will assess drug safety and tolerability in young healthy volunteers, a process that could take a year. If successful, the phase II and III studies would include patients with either Alzheimer's disease or schizophrenia and could take three-five years to complete.