Human pluripotent stem cell based therapy of blood disorders

Funding Type: 
Early Translational IV
Grant Number: 
ICOC Funds Committed: 
Public Abstract: 

Recent technical advancements in human embryonic stem cell (ESC) and induced pluripotent stem cell (iPSC) production have revolutionized their potential applications in regenerative medicine. However, a remaining hurdle in this process is the need for efficient, effective, and stable generation of specific cell types from such stem cells for therapeutic use. The ultimate goal of the proposed study is to develop a novel cell therapy approach to increase the production of therapeutically useful blood cells from human PSCs, especially from patient-specific and genetic mutation corrected iPSCs. Currently, bone marrow transplantation is the best way to cure many blood-related disorders, such as sickle cell anemia, thalassemia, aplastic anemia and certain leukemias. Furthermore, blood transfusion is an effective way to rapidly counteract blood cell loss due to ablative treatments, such as chemotherapy and radiation therapy. Unfortunately, the limiting factor in transplantation and transfusion treatments is the lack of matched donors. The ability to produce unlimited numbers of blood stem cells and/or functioning differentiated blood cells from human ESCs and patient-derived iPSCs will greatly improve availability of such treatments. In this application, we propose to develop stem cell therapies that enhance production of functional blood cells from human iPSCs via delivering cell membrane penetrating factors in cell culture medium. This is highly relevant to the main goals of CIRM.

Statement of Benefit to California: 

Thousands of Californians are suffering from blood-related diseases that may potentially be cured with bone marrow transplantation and/or blood transfusion. However, these life-saving measures are limited by a lack of eligible donors and the necessity of finding correctly matched blood products. Current treatments for some of these conditions can cost patients tens of thousands of dollars per year. Despite these treatments, many patients die from their disease waiting for a bone marrow transplant. Recent technical advancements in human pluripotent stem cells (PSCs) have revolutionized their potential applications in regenerative medicine and have provided enormous hope for these patients. We propose to develop a novel approach to enhance the specificity and efficiency in the production of therapeutically useful blood cells from human PSCs. Therefore, one long term benefit of the proposed work is to improve the treatment of thousands of Californian patients who need to receive healthy, functioning blood cells to alleviate their disease conditions. In turn, this will benefit California’s financial status in reducing the cost of treating these patients with expensive yet ineffective methods. Furthermore, the proposed research will continue to maintain California’s leadership in the field of stem cell research to provide training and education of some of California’s bright young minds.