Principal Investigator:
Joseph M. Scandura, Associate Professor of Medicine
Background & Unmet Need
- Hematopoietic stem cells (HSCs) play an essential role in promoting the lifelong production of mature blood cell lineages
- Stem cell transplantation (SCT) using donor-derived HSCs is an effective therapy to treat multiple hematological malignancies and cancers, including leukemia and myeloma
- However, current SCT methods suffer from limitations in effectively processing an adequate number of HSCs
- Additionally, present technologies are incapable of maintaining or expanding human HSCs ex vivo
- Unmet Need: There is a need for methods that effectively expand HSCs for use in SCT therapies
Technology Overview
- The Technology: Cornell inventors have identified several methods that allow for robust ex vivo expansion of HSCs for research and clinical use
- PoC Data: Human-derived HSCs cultured under hypoxic conditions (1% O2) demonstrated increased expression of CD34+ cells, a phosphoglycoprotein which is used clinically to quantify HSCs levels (see Figure 1A)
- Co-culturing HSCs with endothelial feeder cells (ECs) also increased CD34+ expression in addition to similar markers of HSC levels (CD45, iHSC), see Figure 1B
- Treating cell cultures with N-acetyl cysteine (NAC), an antioxidant that reduces cellular damage from reactive oxygen species, likewise enhances expression of markers of HSC availability (see Figure 1C)
Technology Applications
- Described methods effectively enhance HSC expansion and may increase efficacy of SCT therapies
- Methods may also be used to expand HSCs for research purposes
- Ability to maintain viable HSCs ex vivo affords ability to modify the cells (e.g., genetic manipulations)
Technology Advantages
- Multiple methods to expand HSCs may be used in isolation or in synchrony to enhance HSC expansion effects
- Described culturing conditions are easy to implement and produce robust effects on HSC expansion
Publications
Resources
Intellectual Property
Patents
- US Application US20230212516A1: "Methods for expanding hematopoietic stem cells"
- EP Application EP4158001A1: "Methods for expanding hematopoietic stem cells"
Cornell Reference
- 9494
Contact Information
For additional information please contact
Brian Kelly
Director, Business Development and Licensing
Phone: (646) 962-7041
Email: bjk44@cornell.edu