Blood/Hematopoietic Stem Cells (HSC)
The first stem cell therapy was performed more than 50 years ago using stem cells in the bone marrow that form the blood and immune system. After more than half a million people have undergone stem cell transplantation, the study of bone marrow stem cells continues to guide the science of stem cell biology and lead to innovative cures through gene and cell therapy. Reflecting the importance of this field in stem cell research, the BSCRC has established a large and vital research program in blood, or hematopoietic, stem cells. Our research program encompasses basic, translational and clinical studies ranging from how embryonic stem cells can be coaxed to make hematopoietic cells and restore the immune system, to understanding how stem cells from umbilical cord blood and bone marrow can be best used for transplantation and gene therapy.
Just one example of our efforts in bench to bedside research is the development of gene therapy for the treatment of Sickle Cell disease. Sickle Cell Disease affects one in 500 African Americans and one in 1,000-1,400 Latinos. The disease results from an inherited mutation in the hemoglobin gene that causes red blood cells to "sickle" or become crescent shaped rather than round, making it difficult to pass through small blood vessels. Faculty at UCLA seek to treat sickle cell patients by transplanting the patients with their own bone marrow, using adult blood stem cells that are genetically corrected by adding a hemoglobin gene that blocks the "sickling" of the red blood cells. CIRM recently awarded BSCRC member Donald Kohn, MD, approximately $10 million to develop a clinical trial using this method. This approach has the potential to permanently cure sickle cell disease with significantly less toxicity than with a bone marrow transplant from a donor and would make treatment available to sickle patients who do not have a suitable matched bone marrow donor.
A second area of great potential is our work in the area of leukemia and cancers of the blood. Faculty at UCLA with expertise in blood development focus their research on understanding the mechanisms that control the development and differentiation of blood stem cells, and how their regulation becomes disrupted in diseases like leukemia. Recent discoveries at UCLA have established that blood stem cells originate and are nurtured in the placenta, allowing researchers to mimic their development in cell culture, growing them for use in treating diseases like leukemia.