Multimodal hiPSC-Based Engineering Approach To Restoring Function After Spinal Cord Injury

Funding Type: 
Early Translational IV
Grant Number: 
TR4-06888
Investigator: 
ICOC Funds Committed: 
$0
oldStatus: 
Closed
Public Abstract: 
Spinal cord injury (SCI) affects 11,000 Americans/year & leaves >250,000 individuals with untreatable life-long disabilities. The economic cost of both acute & chronic care for these patients, as well as the loss of the productivity of typically young wage-owners & many of their family members (who must serve as caretakers), makes attempting to remediate this condition not only a humanitarian goal but also a national imperative. Clinically, recovering even 1-2 segments of function would be impactful & alleviate many of the medical complications that characterize SCI. This project is designed to test a multi-modal therapeutic approach to SCI that aims to modify the injured environment of the spinal cord (SC) to both promote repair & blunt those processes that inhibit repair. We also think we can also address certain therapeutic challenges that are unique to SCI -- e.g., loss of tissue & connectivity, cyst formation, scarring, vascular & neural disruption. We will marry stem cell biology to tissue engineering & gene therapy. Furthermore, we will test first in a clinically-relevant animal model, as proof-of-concept, human neural stem cells derived from human induced pluripotent stem cells (hIPSCs), recognizing that, ultimately, a patient’s own immunocompatible hIPSCs derived from his/her own cells should be used to avoid immunosuppressants. At completion of this study, we hope to begin IND-enabling studies.
Statement of Benefit to California: 
Spinal cord injury (SCI) affects 11,000 Americans/year & leaves >250,000 individuals with untreatable life-long disabilities. The economic cost of both acute & chronic care for these patients, as well as the loss of the productivity of typically young wage-owners & many of their family members (who must serve as caretakers), makes attempting to remediate this condition not only a humanitarian goal but also a state and national imperative. Being able to reduce the degree of disability – even recouping a few segments of spinal cord function (e.g., bladder control, use of the thumb, spontaneous respirations) could make a vast humanitarian, social, and economic impact on entire families – not just the patient -- and hence the California economies. We wish to bring an approach to clinical practice that we feel may impact not only recently injured patients but, perhaps, even those who have been in wheel chairs a bit longer.

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