Children Can be Cured of Sickle Cell Disease and Thalassemia After Sibling Cord Blood Transplantation: Results from ViaCell and Children's Hospital & Research Center Oakland
ViaCell, Inc. (Nasdaq: VIAC) and Children’s Hospital & Research Center Oakland reported results today that children with Sickle Cell Disease and Thalassemia can be cured with umbilical cord blood from a compatible sibling. At the Sickle Cell Disease Association of America and National Institutes of Health (NIH) 35th Annual Convention, Dr. Mark Walters, Director of the Blood and Marrow transplant program at Children’s Hospital & Research Center Oakland presented research data demonstrating that cord blood from a relative can be an effective source of stem cells for transplantation in children affected with Sickle Cell Disease and Thalassemia and may have advantages over bone marrow transplantation.
“Patients with Sickle Cell and Thalassemia often lead debilitating lives,” said Dr. Walters. “Through continued research and transplant success, sibling umbilical cord blood has proven to be effective in curing children of these blood disorders. I expect the use of umbilical cord blood will continue to increase and as we gain more experience using cord blood stem cells in transplant medicine, I believe it could outpace the use of bone marrow in transplant medicine.”
The data presented at the Sickle Cell Disease Association of America and NIH meeting showed outcomes from children treated under The Sibling Connection Program, a directed sibling transplant program implemented by ViaCord and Children’s Hospital Oakland Research Institute (CHORI), the research arm of Children’s Hospital & Research Center Oakland. This program has resulted in cord blood treatments in more than 100 children to date. Of the children treated under the Sibling Connection Program, 17 were transplanted for Sickle Cell Disease and 23 were transplanted for Thalassemia. The median age of patients treated for Sickle Cell Disease was 8 years and 5 years for patients treated for Thalassemia.
Transplantation of sibling umbilical cord blood has demonstrated clinical advantages over bone marrow transplantation in young children. In particular, the risk of graft-versus-host (GvHD) disease, a common side-effect and the leading cause of death in transplant medicine, is reduced. Of the children treated, six patients with Sickle Cell Disease had acute GvHD. No patients treated for Sickle Cell Disease had chronic GvHD. In addition, no acute or chronic GvHD was observed in patients transplanted for Thalassemia.
The median time to neutrophil recovery (ANC greater than 500 cells per microliter) and platelet recovery (greater than 20,000 per microliter) in patients treated for Sickle Cell Disease was 18 days and 36 days, respectively. 82% of the patients treated for Sickle Cell Disease survive and are disease-free. The median time to neutrophil recovery (ANC greater than 500 cells per microliter) and platelet recovery (greater than 20,000 per microliter) in patients treated for Thalassemia was 25 days and 47 days, respectively. 96% of the patients treated for Thalassemia survive and 91% are disease-free.
In 2006, ViaCell and CHORI combined their efforts in the area of directed transplants for sibling donor umbilical cord blood to form the Sibling Connection Program. To date, over 100 children have been treated by cord blood from units collected and processed through this program. This includes transplants through cord blood collected, preserved and stored with ViaCord and transplants using cord blood stored through CHORI's Sibling Donor Cord Blood Program. The Sibling Connection Program provides ViaCord’s comprehensive cord blood collection, processing and five years of storage at no cost to families who have a child diagnosed with a condition that can be treated with cord blood stem cell transplant and meet the other requirements of the program.
About Sickle Cell Disease
Sickle Cell Disease is an inherited blood disorder where red blood cells are sickle or crescent shaped. The abnormally-shaped cells become rigid and prevent normal flow of oxygen to tissues, causing tissue damage. Common symptoms include chest pain, frequent infections, jaundice, and anemia. There is currently no universal cure for Sickle Cell Disease. Complications of Sickle Cell Disease can be treated with antibiotics, pain management, intravenous fluids, blood transfusions and surgery. Over 2.5 million people in the United States carry the trait and over 80,000 have sickle cell anemia. Sickle Cell Disease predominantly affects African Americans and people of Mediterranean descent. September is National Sickle Cell Awareness Month.
Umbilical cord blood has been successfully transplanted in patients with Sickle Cell Disease and Thalassemia as an emerging therapeutic treatment option. ViaCord released its first cord blood unit from a related source to treat Sickle Cell Disease in 2000. 30% of all enrollments in the ViaCord/CHORI Sibling Connection Program are with families who have children affected with Sickle Cell Disease.
About Thalassemia
Thalassemia is a hereditary blood disorder characterized by decreased production of hemoglobin, a critical oxygen-carrying protein in red blood cells. This results in anemia and shortage of red blood cells. Diagnosis is typically early in childhood with lifelong red blood cell transfusions and resulting complications. The symptoms of thalassemia depend on the type and severity of the disease and include anemia, jaundice, enlarged spleen and liver, abnormal facial bones and poor growth. It is estimated that over 2 million people in the United States carry the genetic trait for Thalassemia and approximately 1,000 people are living with Thalassemia in the U.S.
About Umbilical Cord Blood
Umbilical cord blood is a valuable, non-controversial source of stem cells with proven therapeutic effect in treating over 40 diseases. These diseases include cancers such as Acute Lymphoblastic Leukemia (ALL) and Non-Hodgkin’s lymphoma, certain bone marrow failure syndromes such as severe aplastic anemia and Diamond Blackfan anemia, certain blood disorders such as sickle cell anemia, thalassemia and other genetic disorders. Over 8,000 cord blood transplants have been performed worldwide. Studies have shown that umbilical cord blood transplants from a family member, rather than from a non-relative, have a significantly higher survival rate than transplants from an unrelated donor.
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