Bone marrow and blood stem cell transplantation is a potentially curative treatment for patients with a variety of blood-related disorders, including leukemia, lymphoma, anemias and autoimmune diseases, as well as non-blood disorders such as cancer and enzyme deficiencies. Survival and quality of life of patients with these disorders have been improving steadily since bone marrow transplantations, containing blood stem cells, came into clinical use in the 1970’s. Now among the few routinely performed cell-based therapies, bone marrow transplantation thus serves as an important paradigm for clinical application of other types of stem cells. Although routinely used to treat a variety of diseases, the pretreatment preparing the patient for transplant is grueling and the risk of life threatening complications are unacceptably high.
The research in this proposal is intended to understand how blood stem cells mature into different cell types. We are investigating the differentiation pathways of normal hematopoiesis and are comparing these to conditions of hematopoietic stress. The goal is to be able to detect, prevent and treat blood-related disorders and to improve the efficiency and safety specifically of blood stem cell transplantation. With increased safety of transplantation regimens, this type of treatment will become available to a greater number of patients and lead to higher survival rates and fewer complications. Stem cell transplantation may also eventually be used to treat non-life-threatening, but burdensome, disorders.
In addition, this research will contribute to the California education and health care systems by training undergraduate, graduate and postdoctoral students into highly skilled stem cell biologists.