The Duke University School of Medicine has received approval from the Food and Drug Administration to market DUCORD, a product derived from umbilical cord blood stem cells collected by the Carolinas Cord Blood Bank, for use as an approved treatment of patients with various blood disorders.
The FDA’s approval of Duke’s cord blood stem cells for use in treating blood disease indicates that the CCBB’s umbilical cord stocks are of sufficient quality. The approval means procedures involving these cells do not need to be listed as experimental. This endorsement is indicative of the quality of Duke treatments derived from umbilical cord stem cells, and it makes further research into cord blood stem cell therapies more likely to receive regulatory approval.
“This is a huge milestone and a statement by the FDA about the place that cord blood stem cells have in this research,” said Dr. Robert Califf, director of the Duke Translational Medicine Institute.
Califf added that FDA approval was a strong indication of the consistency of the CCBB’s treatments.
The National Institutes of Health provided a grant to found a bank for umbilical cord stem cells at Duke in 1998. Since that time, the CCBB has been collecting donations of cord blood from mothers of healthy babies delivered at various affiliated hospitals, including Duke Hospital, Durham Regional Hospital and the University of North Carolina at Chapel Hill Hospital. Such blood is rich in hematopoietic stem cells, which are responsible for the manufacture of red and white blood cells. Duke’s medical school has designed an injectable product from these cells that can be used to regenerate a patient’s hematopoietic system, filling a role traditionally occupied by bone marrow transplants.
“Cord blood stem cells are of proven benefit in patients with disorders that prevent them from making their own normal blood cells either because of a disease process—including genetic enzyme deficiencies and immune disorders—or because of treatment for cancer,” Dr. Nancy Andrews, dean of the the medical school, wrote in an email Wednesday.
All cord blood collected by the CCBB is registered on the National Marrow Donor Program Registry, which vastly increases the odds of a patient finding a compatible cell match. Until now, Duke could not market such treatments as being commercial quality. With FDA approval, however, Duke joins only a handful of programs that are past the experimental phase. This rare licensure gives the quality of CCBB stem cells a high rating for hematopoietic procedures, but it also has implications for other ongoing research.
Dr. Joanne Kurtzberg, medical director of CCBB, remarked that both Duke’s research and treatment into other applications of umbilical cord stem cells may benefit from FDA licensure.
“By having a licensed product, derivative products that we manufacture from the same cord blood will have a better chance of going through the regulatory pathway and also becoming approved products,” Kurtzberg said.
Andrews noted that cord blood stem cells may have applications that are yet to be discovered.
“Much of the excitement will come from finding out what other types of diseases may be treated using cord blood stem cells,” Andrews said.