CHILDREN'S NATIONAL MEDICAL CENTER
Children’s sees more than 8,000 children per year and treats more children with rare metabolic diseases than any hospital in the world. Our primary collaborator, Marshall Summar, M.D., is Chief of Genetics and Metabolism at Children’s. Dr. Summar is an internationally recognized disease expert who has introduced us to a broad network of disease experts and major transplant centers. Dr. Summar serves as Chairman of the National Organization for Rare Diseases and works closely with the NIH and FDA on rare disease initiatives. The combination of Children's deep knowledge of rare disease genetics and access to patients coupled with our ability to re-create rare disease biology has the potential to discover therapies for rare children’s diseases that have no approved treatments. We are currently collaborating with Children’s to discover treatments for propionic acidemia and methylmalonic acidemia.
ARUN SANYAL, M.B.B.S., M.D.
Our collaborator and consultant in NASH, Dr. Arun Sanyal, is the Charles Caravati Professor and chair of the Division of Gastroenterology, Hepatology and Nutrition, in the Department of Internal Medicine at Virginia Commonwealth University. Dr. Sanyal currently leads an FDA committee working on defining clinical endpoints and regulatory approval criteria for NASH and is similarly working with the European Medicines Agency. He is also Chairman of the NIH NASH Research Network, serves as principal investigator for a number of NASH clinical studies, and consults for a number of pharma companies that are in the latter stages of NASH drug development.
The National Cancer Institute is funding our development of physiological models of the tumor microenvironment. We are collaborating in this effort with the University of Virginia to continue development and validation of our models.