Ex-Vivo Method to Maintain Hematopoietic Stem Cells

An efficient way to improve outcomes of bone marrow and umbilical cord blood transplants to increase survival in blood cancer patients. The invention works by expanding hematopoietic stem cells in culture using a defined medium with specific inhibitor combinations. 

Problem: 

Expanding hematopoietic stem cells (HSCs) is paramount in stem cell research and for clinical use of HSCs. However, HSC homeostasis is not well understood and methods for HSC expansion have so far required the use of complex and expensive cocktails of cytokines and/or support cells. 

Solution: 

Dr. Klein and his team developed a method to expand and maintain HSCs ex-vivo using available GSK3 and mTOR inhibitors potentially enabling production of a greater number of long term HSCs without the use of exogenous hematopoietic cytokines and other factors. This combination, in mice, increases the number of HSCs, improves the frequency of engraftment, and shortens the time to hematopoietic recovery in transplant recipients. Additionally, these novel culture conditions offer an excellent research tool to explore HSC biology.

Advantages: 

• Maintenance and expansion of HSCs without inducing cell lineage commitment
• Maintenance and expansion of HSCs without the need for cytokines
• Use of clinically approved drugs

Applications: 

• Pre-treatment to increase efficacy of bone marrow transplantation
• Pre-treatment to increase efficacy and survival rate of UCB transplantation
• Potential use to expand and maintain other stem cells
• Potential research tool to expand HSCs that may be applied to other adult stem cells 

Stage of Development: 

• Ex-vivo maintenance of HSCs, including cord blood HSCs
• In-vivo data showing improved survival of recipients who received HSCs cultured in inhibitor combination. 

Intellectual Property: 

• US 9,050,315
• US 9,452,187 

Reference Media: 

• Huang, J. et al.; J Clin Invest, 2009, 119(12): 3519.
• Huang, J. et al.; Nat Med, 2012, 18(12): 1778.

Desired Partnerships: 

• License
• Co-development  

Docket # W5373