The research team led by Dr Lakshmanan Krishnamurti, a pediatric hematologist/oncologist at Children's Hospital developed a new approach of bone marrow transplantation which relies on reduced-intensity conditioning (RIC).
RIC regimens are less toxic to patients and therefore can be offered to patients with severe sickle cell disease because they eliminate life-threatening side effects generally associated with bone marrow transplantation.
"Bone marrow transplant is the only known cure for sickle cell disease. But doctors have avoided performing them in these patients because complications from a traditional bone marrow transplant can be life-threatening," said Dr. Krishnamurti, director of the Sickle Cell Program at Children's Hospital.
"Through the reduced-intensity approach we developed, the potential for complications is dramatically lessened.
"This study offers hope for a cure to thousands of patients with severe sickle cell disease," he added.
Traditionally, bone marrow transplants require heavy doses of chemotherapy prior to transplant in order to destroy the recipient's bone marrow so it will not reject the donated marrow. But with their bone marrow destroyed, transplant recipients become vulnerable to life-threatening complications.
The results are published in Biology of Blood and Marrow Transplantation.