According to a story from bioportfolio.com, The California Institute for Regenerative Medicine has given $5.74 million to the City of Hope, a private, nonprofit clinical research center that is primarily known for its development of cancer treatments, although it has also conducted work to treat other condition as well. The funding is planned to be used for a stage I clinical trial of a new blood stem cell transplant procedure meant to treat adults with severe cases of sickle cell disease.
Sickle cell disease is an inherited blood disorder characterized by blood cells that are an unusual, sickle shape. The most severe and common form of sickle cell disease is sickle cell anemia; sickle cells do not survive as long as regular blood cells, causing low cell counts. Problems tend to begin at around 6 months old, and it is caused by an hemoglobin abnormality. In an affected person, hemoglobin A, which is the typical oxygen carrier in human blood, is partially replaced with hemoglobin S, which causes the misshapen cells to form. These cells easily clog blood vessels, and sickle cell disease can lead to many dangerous complications. Some symptoms include severe bouts of pain (sickle-cell crisis), swelling of the feet and hands, vulnerability to bacterial infection, and stroke. It is more common in people of African ancestry. Treatment includes infection prevention such as antibiotics and vaccines, high intake of fluids, pain medication, and folic acid supplementation. People with sickle cell disease have a life expectancy of 40 to 60 years. To learn more about sickle cell diseases, click here.
The procedure involves transplanting stem cells that produce blood from one person to a patient that has received a mild chemotherapy treatment that is meant to eliminate the majority of bone marrow stem cells affected by disease from the patient. More unusually, the donor’s stem cell samples are stripped of CC4+ T immune cells before the transplant. This precaution is meant to prevent the possibility of graft-versus-host disease so the transplanted cells can begin producing healthy blood cells.
The City of Hope has already performed similar transplant operations previously, but the procedure is typically reserved for children. The treatment is limited by the need for a genetically matched donor, but researchers are confident that testing the procedure for adults could ultimately help many people with severe sickle cell disease, and offers a curative option for treatment.
