Development of pluripotent-derived hNSC candidate cells for the treatment of chronic SCI

Funding Type: 
Early Translational III
Grant Number: 
TR3-05575
Investigator: 
ICOC Funds Committed: 
$0
oldStatus: 
Closed
Public Abstract: 
Multipotent human neural stem cells (hNSC) have shown potential for the treatment of disease and injury in the brain and spinal cord. hNSC can be derived from adult or fetal tissue, as well as from pluripotent cells. Cells have surface markers that can be selected for or against by cell sorting techniques. Selecting fetal hNSC for the cell surface marker CD133, and against the cell surface marker CD34, enriches for cells that can survive, migrate, and repair the injured spinal cord. Experiments testing fetal hNSC selected for these markers have led to several clinical trials in the USA, as well as a trial for chronic spinal cord injury approved in Switzerland and ongoing at the University of Zurich. In addition, selection for these markers may reduce the potential for tumor formation by pluripotent-derived hNSC. As banks of pluripotent human embryonic stem cells are developed for to allow donor-host immunological matching, and the technologies for individual patient-derived human induced pluripotent cell therapies are realized, strategies to enhance safety and consistency, such as selecting for cell surface markers, may enable translation of these pluripotent cells. However, sorting human pluripotent stem cells for these cell surface markers has not been tested. In this proposal, we seek to develop clinically compliant GMP grade pluripotent-derived hNSC selected for these surface markers, and test their efficacy and safety in the treatment of chronic spinal cord injury.
Statement of Benefit to California: 
Multipotent human neural stem cells (hNSC) have shown potential for the treatment of disease and injury in the brain and spinal cord. hNSC can be derived from adult or fetal tissue, as well as from pluripotent cells. Cells have surface markers that can be selected for or against by cell sorting techniques. Selecting fetal hNSC for the cell surface marker CD133, and against the cell surface marker CD34, enriches for cells that can survive, migrate, and repair the injured spinal cord. Experiments testing fetal hNSC selected for these markers have led to several clinical trials in the USA, as well as a trial for chronic spinal cord injury approved in Switzerland and ongoing at the University of Zurich. In addition, selection for these markers may reduce the potential for tumor formation by pluripotent-derived hNSC. As banks of pluripotent human embryonic stem cells are developed for to allow donor-host immunological matching, and the technologies for individual patient-derived human induced pluripotent cell therapies are realized, strategies to enhance safety and consistency, such as selecting for cell surface markers, may enable translation of these pluripotent cells. However, sorting human pluripotent stem cells for these cell surface markers has not been tested. In this proposal, we seek to develop clinically compliant GMP grade pluripotent-derived hNSC selected for these surface markers, and test their efficacy and safety in the treatment of chronic spinal cord injury.

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