Baylor College of Medicine has teamed up with the National Urea Cycle Disorders Foundation (NUCDF), a leading patient advocacy organization, to advance research into understanding, treating and even curing urea cycle disorders, caused by a genetic defect that results in a deficiency in an enzyme key to the cycle’s proper function.
The urea cycle is responsible for detoxifying ammonia – a byproduct of protein metabolism – in the bloodstream. Normally, the cycle converts ammonia in the bloodstream to urea, which is then eliminated. In urea cycle disorders, ammonia builds up to toxic levels that, without intervention, damage the brain and can quickly cause coma and ultimately death.
Supported by NUCDF, the CureUCD Center for Preclinical Therapeutic Discovery will be established at Baylor to engage technology and drug companies in early stage testing and validation studies of new small molecules, nucleic acids, as well as cell and gene therapies for these disorders.
“Great progress has been made in the clinical research and infrastructure required for testing cures for urea cycle disorders,” said Dr. Brendan Lee, the Robert and Janice McNair Endowed Chair and Professor, and chair of molecular and human genetics at Baylor, who will lead the Center. “There is still no cure despite significant improvements in care and quality of life. We need to find a cure. This new collaboration will jump start that.”
“We are very excited about this model partnership to establish the CureUCD Center for Preclinical Therapeutic Discovery to catalyze development and help drive new treatments for UCD through the pipeline,” said Cindy LeMons, executive director of the Foundation. “Our children and adults with UCD are in a fight for their lives. We are laser focused on finding a cure.”
Established in 1988, the National Urea Cycle Disorders Foundation is the only nonprofit organization in the world solely dedicated to saving and improving the lives of children and adults with urea cycle disorders by supporting critical research, providing education and information to families and medical professionals, and raising public awareness so no child or adult suffers or dies undiagnosed.