Large-Scale Adult Stem Cell Trials Planned for Children With Sickle Cell Anemia

Washington University Press Release, July 11, 2008

In an article published in the August 2007 issue of the Journal of Bone Marrow Transplantation, Dr. Shalini Shenoy reviewed the latest advancements in stem cell transplantation which allow for low toxicity and high success rates in the treatment of sickle cell anemia. However, as Dr. Shenoy pointed out, stem cell therapies are not yet in common use for the treatment of this disease. Today, Dr. Shenoy is now leading one of the largest studies ever to be conducted in the treatment of sickle cell anemia, as this comprehensive clinical trial is designed specifically to test the safety and efficacy of stem cell therapy on a grand scale. As associate professor of pediatrics at the Washington University School of Medicine, and medical director of the pediatric bone marrow transplant program at St. Louis Children's Hospital, Dr. Shenoy is directing the nationwide, multicenter Phase II clinical trials for the use of adult stem cell therapy in the treatment of children with the most advanced forms of the disease. Qualifying participants will receive stem cells derived either from bone marrow or umbilical cord blood. A total of 45 patients are sought, between the ages of 3 and 16, who suffer from the most severe, most life-threatening forms of sickle cell anemia.

According to Dr. Shenoy, "Right now, blood stem cell transplant is the only potential curative therapy for severe sickle cell disease." Approximately ten patients with sickle cell anemia improved after receiving stem cell transplants in preliminary trials, the results of which were so positive that interest was generated in expanding the trials to a larger population group.

An inherited blood disorder in which hemoglobin is abnormally shaped, sickle cell anemia afflicts approximately 70,000 people in the U.S. alone, occurring in 1 in every 500 African-American births, and 1 in approximately every 1,200 Hispanic-American births. Previously, blood transfusions and bone marrow transplants have offered the only known treatments, both of which have serious complications which include graft-versus-host disease and a 10% mortality rate from bone marrow transplantation. By contrast, hematopoietic stem cell therapy has been shown to restore normal hematopoiesis to people who are suffering with sickle cell anemia, with very low risks.

The upcoming nationwide trial is supported by the National Marrow Donor Program, the Sickle Cell Disease Clinical Research Network, the Bone Marrow Transplant Clinical Trials Network of the National Heart, Lung and Blood Institute, and the Pediatric Blood and Marrow Transplant Consortium.