Public Release: 

NHLBI Cord Blood Transplantation Study Begins

NIH/National Heart, Lung and Blood Institute

The first multicenter study of umbilical cord blood transplants from unrelated, newborn donors was announced today by the National Heart, Lung, and Blood Institute of the National Institutes of Health.

The 5-year, $30 million study is designed to show whether cord blood transplantation is a safe and effective alternative to bone marrow transplantation for children and adults with a variety of cancers, blood diseases, and genetic disorders.

Said NHLBI Director Dr. Claude Lenfant, "Although earlier NHLBI studies have provided evidence that cord blood transplantation can be used for children with some cancers and genetic disorders, a myriad of scientific and ethical questions related to this procedure remain. We hope that this study will provide the answers to these questions."

Bone marrow transplantation is an effective therapy for a variety of genetic disorders and blood diseases. However, for patients who don't have a matching related donor, the current system for finding a matching unrelated donor can be a long, frustrating, and ultimately unsuccessful process. This is especially true for people from ethnic and racial minorities.

Human umbilical cord blood is a rich source of the stem and progenitor cells that are present in bone marrow, and cord blood from related donors has been successfully transplanted in many children worldwide. In addition, cord blood from unrelated donors has been successfully transplanted in 51 children in two recent U.S. studies. The cord blood units for both studies was provided by the New York Blood Center, which was funded by the NHLBI.

The new study will be conducted at three facilities that will be responsible for collecting the cord blood units and at seven centers that will perform the transplants. A data coordinating center will identify the cord blood units for transplant, as well as collect the data from the study.

The study will transplant four groups of 50-75 patients each: adults weighing more than 88 pounds (40Kg), children weighing less than 88 pounds (40 Kg), patients with cancers, and patients with other conditions currently treatable with bone marrow transplantation. The transplant centers will use standardized protocols for enrolling patients, preparing them for transplant, and treating them after the transplant.

The participating transplant centers are the Dana Farber Cancer Center, Boston; the Fred Hutchinson Cancer Research Center, Seattle; the University of California, Los Angeles (UCLA). Children's Hospital of Orange County, Orange, CA; Indiana University, Indianapolis; Duke University Medical Center, Durham, NC; and the University of Minnesota, Minneapolis.

The collection and storage centers are located at Children's Hospital of Orange County, Duke University, and UCLA.

The coordinating center is the Emmes Corporation, Potomac, Maryland.

For more information, contact the NHLBI Communications Office, 301-496-4236.

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