A compound developed at the Milwaukee Institute for Drug Discovery (MIDD) at the University of Wisconsin-Milwaukee (UWM), known as GL-Damonia, has entered Phase 1 clinical trials at the U.S. Food and Drug Administration (FDA). This marks a crucial step toward potentially making the drug available for patient treatment for depression and Alzheimer's disease. The drug was co-developed by Jim Cook, an emeritus adjunct professor in UWM’s Chemistry & Biochemistry Department and one of the founders of MIDD, and Etienne Sibille at the University of Toronto.
Novel Approach to Neurodegenerative and Psychiatric Disorders
GL-Damonia is a derivative of benzodiazepines, a class of drugs that includes common anxiety medications like Valium and Xanax. However, GL-Damonia is designed to avoid the adverse side effects associated with these drugs, such as sedation, amnesia, and addiction. Cook and his team aimed to target different receptors in the brain to achieve therapeutic benefits without these drawbacks.
Cook explains, "If you take a young mouse and let it live for six months, what happens is the neurons, the axons and dendrites and synapses (brain cells) start to die. You give them my drug, and they start to grow back, almost immediately to where they were in a young mouse."
Preclinical Promise
Preclinical studies have demonstrated promising results. In animal models of depression, GL-Damonia administration led to the regrowth of shortened dendrites and axons, effectively returning the mice to a baseline state without depression symptoms. These findings suggest that GL-Damonia could potentially reverse the neuronal damage associated with both aging and depression.
Phase 1 Trial Objectives
The FDA Phase 1 trials will primarily focus on assessing the safety and tolerability of GL-Damonia in a small group of healthy volunteers. Researchers will monitor participants closely to identify any potential side effects and determine a safe dosage range. If the drug demonstrates a favorable safety profile, it will proceed to Phase 2 trials, where its efficacy in treating depression will be evaluated in a larger patient population.
Future Development and Potential Impact
While Sibille's primary interest lies in treating depression and Alzheimer's disease with GL-Damonia, Cook is optimistic about its potential application in treating schizophrenia as well. The progression of GL-Damonia through clinical trials represents a significant advancement in the search for more effective and safer treatments for these debilitating conditions. Although the success rate for drugs entering clinical trials is low (less than 10% make it through all three phases), the potential rewards are substantial. Successful development and commercialization of GL-Damonia could provide significant financial returns to UWM, the UWM Foundation, and MIDD, while also fulfilling the researchers' primary motivation: to improve patient outcomes and quality of life.
