Children's National's Urea Cycle Disorders Consortium Foresees Innovation from Major NIH Funding

WASHINGTON, Oct. 10, 2014 /PRNewswire-USNewswire/ -- Children's National Health System's  Urea Cycle Disorders Consortium was awarded $6.25 million over the next five years in the latest round of funding  from the National Institutes of Health (NIH) to perform clinical research and develop new treatments for patients with urea cycle disorders (UCD), rare but devastating genetic conditions.

Mark L. Batshaw, MD,  Physician-in-Chief and Chief Academic Officer at Children's National, is the principal investigator for the program. Mendel Tuchman, MD,  Chief Research Officer and Scientific Director of the Children's Research Institute, is the administrative director and co-principal investigator of the Urea Cycle Disorders Consortium.  Marshall Summar, MD,  Chief of Genetics and Metabolism at Children's National and Ms. Cynthia LeMons, President of the National Urea Disorders Foundation  are co-principal investigators. 

This week's funding announcement of the 2014 Rare Diseases Clinical Research Network  (RDCRN) consortia and data management coordinating center marked the third five-year funding cycle for the Urea Cycle Disorders Consortium with Children's National as its lead institution.  Since its inception in 2003, the Urea Cycle Disorders Consortium (UCDC) has also received matching philanthropic support from the O'Malley Family Foundation, the Kettering Fund, the Dieter-Hopp Foundation, and the Rotenberg Family Foundation.

"The Consortium has assisted in bringing three new drugs to market to treat these devastating disorders that primarily affect children, leading to brain injury and often death," says Dr. Batshaw.  

There are several thousand rare diseases affecting 25 million Americans, but only a few hundred diseases have any treatments available. Dr. Tuchman indicated that "the creation and funding of the UCDC has been the culmination of many years of clinical and research work by our group to improve the lives of children and adults with rare metabolic disorders."

With the new award, scientists at the consortia will test new therapies and continue to conduct a longitudinal natural history study of affected patients to better understand the course of the disorders.  The consortium has more than 650 participants enrolled in studies, ten times more than any previous study conducted in this group of rare disorders, Dr. Batshaw says.

About Children's National Health System
Children's National Health System, based in Washington, DC, has been serving the nation's children since 1870. Children's National's hospital is Magnet® designated, and is consistently ranked among the top pediatric hospitals by U.S.News & World Report. Home to the Children's Research Institute and the Sheikh Zayed Institute for Pediatric Surgical Innovation, Children's National is one of the nation's top NIH-funded pediatric institutions. With a community-based pediatric network, eight regional outpatient centers, an ambulatory surgery center, two emergency rooms, an acute care hospital, and collaborations throughout the region, Children's National is recognized for its expertise and innovation in pediatric care and as an advocate for all children. For more information, visit ChildrensNational.org, or follow us on Facebook and Twitter.

SOURCE Children's National Health System