Dec. 10, 2009
Adult stem cell transplants from bone marrow and cord blood have been used successfully in the past to treat children with sickle cell anemia. Results with children, using related donors, have been very encouraging as noted by Bernaudin et al.:
"Allogeneic hematopoietic stem-cell transplantation (HSCT) is the only curative treatment for sickle cell disease"
and by Shenoy in 2007:
"Currently, hematopoietic SCT (HCT)is the only intervention that can restore normal hematopoiesis to provide a cure in sickle cell disease."
But as these authors note, application on a wide scale for sufferers of sickle cell disease has been limited, due to the harshness of the treatment, and more often due to finding a relative for a transplant match (see this video, "My Brother Saved My Life", for one story of success.)
"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."
Further studies have shown that even mismatched umbilical cord blood can provide an effective treatment for children with sickle cell anemia.
NOW a new study indicates that adults can also be successfully treated for sickle cell anemia with adult stem cells. The successful treatment involved a gentler radiation therapy and antibody treatment, and matching of donors. After 30 months, all of the patients are alive, and nine of the ten patients had successful grafts and are considered cured of sickle cell disease, according to the study published in the New England Journal of Medicine.
"It's been transforming for these patients," Tisdale said. "These were the sickest of the sick patients. Some were in the hospital every other week for pain or other crises. Today, some have gone back to school and to work. One patient had a baby."
The results emphasize that adult stem cells provide the only successful stem cell treatments, and also that much more needs to be done to make resources available for adult stem cells, putting the patients first.