Children's National Medical Center immunologist and blood and marrow transplant physician Naynesh Kamani, MD, will serve as the study co-chair for a new national clinical trial of unrelated donor marrow and umbilical cord blood transplants for severe sickle cell disease.
Children's National will join more than 20 other medical institutions in the first-ever Phase II clinical trial of this treatment. If successful, the Sickle Cell Unrelated Transplant (SCURT) trial has the potential to extend a promising and possibly curative treatment option to more severely affected patients. Sickle cell disease affects approximately 70,000 people in the United States.
For many years, doctors have used blood or marrow transplants to treat severe cases of sickle cell disease. However these treatments have been available only to patients with a matched family member to donate blood or marrow stem cells. Only 14 percent of severe sickle cell cases have a matched donor within their family. Successful transplants using unrelated donors could create a viable treatment option for more sickle cell patients.