Making Stem Cell Therapies Safer: Targeting ES Cells with Cell Cycle and miRNA Antagonists

Funding Type: 
Basic Biology III
Grant Number: 
ICOC Funds Committed: 
Public Abstract: 

Stem cell therapies have the potential to result in entirely new treatments for a vast variety of currently incurable diseases. The goal of these therapies is to regenerate lost cells or tissues through the use of established human embryonic stem cell lines or even from the patient's own cells. One major limitation of current stem cell therapy approaches is that they may give rise to cancer. Embryonic stem cells have a limitless potential to reproduce and if they remain in a undifferentiated state could give rise to tumors. This proposal seeks to make stem cell therapies safer by using two novel targets that can selectively kill undifferentiated stem cells while sparing differentiated cells or normal cells. We propose to use small molecule inhibitors of the cell cycle and inhibitors of specific small RNAs to kill embryonic stem cells that have remained in an undifferentiated state. If validated, these therapeutic approaches could be developed to make stem cell therapies safer and hasten stem cell based therapies to the clinic.

Statement of Benefit to California: 

The State of California is committed to developing new approaches to treat currently incurable diseases through the application of stem cell biology technologies. While there is much excitement about the prospects for these new treatments, there is also potential concern, that current strategies may also increase the risk of developing cancer. This proposal seeks to use new strategies to block the growth of embryonic stem cells that pose the risk of developing into cancer. It is hoped that such approaches will make stem cell-based therapies safer for us all. Development of such approaches would be expected to advance the field of stem cell biology and speed delivery of new and safer treatments to the clinic.