Pluripotent cells, specifically human embryonic stem cells (hESCs) and induced pluripotent stem cells (iPSCs), have the potential to differentiate into all of the more than 200 cell types in the body. This furnishes the possibility of providing a large supply of cells to replace or repair damaged or malfunctioning tissues within the body, and thereby opening a new and potentially much more efficacious method for treatment of chronic illness such as cardiovascular, diabetic, and neurodegenerative diseases. However, only differentiated cells can be safely transplanted into the human body. Trace amounts of pluripotent cells resident among the differentiated cells can form teratomas, ill-organized and proliferative cell masses, post-transplantion. Currently, methods to prevent and eliminate teratoma formation are not available. As a result, teratoma formation has become a major roadblock to the clinical usage of pluripotent cell derivatives. The proposed research will study the mechanism of teratoma formation, establish risk evaluation methods, and develop and identify antibodies and small molecules to eliminate and prevent teratoma formation. The results of this research will open the gate to move basic stem cell research into viable stem cell based clinical therapies.
Statement of Benefit to California:
The proposed researches target one of the major issues of stem cell based therapy--teratoma formation. This research:
1). will establish risk evaluation methods for teratoma formation;
2). will establish in vitro teratoma formation model which will greatly facilitate studies of its mechanism;
3). develop antibodies and chemicals that can be used to prevent teratoma formation;
4). develop teratoma formation eliminating methods.
These works will advance all the pluripotent cell based clinical cell therapies. Results from these works will provide commercializable items which can benefit California citizens' economy and health.