Genetic diseases of the blood system range from blood cancers (e.g., leukemias) to autoimmune diseases (e.g., Type 1 diabetes, multiple sclerosis and rheumatoid arthritis). As such, they are amongst the most severe diseases afflicting California and the world as a whole. These genetic blood diseases are caused when our body’s own blood cells go awry.
It has been shown that many types of genetic blood disease can be treated, if not outright cured, by replacing a patient’s faulty blood cells with new, healthy blood cells. Clinical trials have shown encouraging results for patients with various blood cancers and autoimmune disorders when they are injected with new, healthy blood-forming stem cells, thereby generating a new and healthy blood system and replacing their own faulty blood cells. While these results are encouraging, there is a long ways yet to go to make such treatments safer for patients and more commonplace in the clinic.
In the past funding period, we have made significant advances towards this goal of replacing patients’ faulty blood systems with new, stem cell-derived blood systems. Firstly, we have made steps towards generating new human blood stem cells in a dish from embryonic stem cells. The eventual goal of this salient is to mass-producing new blood stem cells in a dish for patients with genetic blood diseases that are in need of them. Secondly, we have identified combinations of biological agents (known as antibodies) that can safely deplete mouse blood stem cells. The eventual goal of this second line of research is to eventually identify combinations of similar biological agents that can safely deplete the faulty blood stem cells in human patients with genetic blood diseases, allowing us to then inject new, healthy blood stem cells to regenerate a healthy blood system.