The Vanderbilt University Medical Center has passed a key hurdle in its quest to develop a treatment for Alzheimer’s disease with the announcement that the U.S. Food and Drug Administration (FDA) has approved a Phase 1 clinical trial of the therapy.
The development marks one of the few times that a university has taken a treatment from discovery to the first clinical trial without the support of a pharmaceutical company.
“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,” Susan R. Wente, PhD, provost and vice chancellor for academic affairs, said in a press release.
“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,” she added.
Researchers want to know whether the compound they developed will selectively activate a key receptor in the brain, as they hope.
“Importantly, at this early stage, the FDA has only granted permission to assess potential safety of this investigational new drug in healthy volunteers,” said P. Jeffrey Conn, PhD, a professor of pharmacology at the Vanderbilt University School of Medicine and director of the Vanderbilt Center for Neuroscience Drug Discovery.
If the drug proves safe, the research team will ask for a clinical trial to see if the compound improves cognition in Alzheimer’s patients.
“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,” Conn said.
“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 clinical testing phase due to lack of funding,” he added.
The feat was made possible by funding from the William K. Warren Foundation, whose philanthropy includes medical research.
“Although this is an important sequential milestone, the only milestone that matters to us is the hope that one day we will learn that this investigational new drug has positively and safely changed the life of a patient suffering from a brain disorder such as schizophrenia or Alzheimer’s disease,” said John-Kelly Warren, the foundation’s CEO.
“That day will warrant a celebration felt in the heavens,” he said. “Until then, we are prepared to support the . . . research team until they can deliver the necessary results.”