Autologous stem cell-derived interneuron cell therapy for spinal cord injury (SCI)

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Grant Award Details

Grant Type:
Quest – Discovery Stage Research Projects
Grant Number:
DISC2-14041
Investigator(s):
Name:
Matthew Goodus
Institution:
BrainXell Therapeutics
Type:
PI

Disease Focus:
Neurological Disorders, Spinal Cord Injury
Human Stem Cell Use:
Embryonic Stem Cell, iPS Cell
Award Value:
$2,025,000
Status:
Active

Grant Application Details

Application Title:

Autologous stem cell-derived interneuron cell therapy for spinal cord injury (SCI)

Public Abstract:
Research Objective

Functional restoration following spinal cord injury using defined excitatory and inhibitory spinal interneuron progenitor cell transplantation

Impact

The development of a stem cell derived progenitor cell therapy with disease modifying potential for spinal cord injury

Major Proposed Activities

  • Define and characterize DI4 GABA and V3 Glut spinal interneuron progenitor cells derived from pluripotent stem cells
  • Evaluate and compare the effects of transplantation of DI4 GABA vs. V3 Glut spinal interneuron progenitors subacutely and chronically after a non-clinical spinal cord injury model
  • Evaluate the effects of combined transplantation of DI4 GABA and V3 Glut spinal interneuron progenitors subacutely and chronically after a non-clinical spinal cord injury model
  • Evaulate the effects of chemogentic activation and inactivation through DREADD receptors expressed on transplanted dI4 GABA vs. V3 Glut spinal interneuron progenitors on behavioral outcomes after SCI
  • Establish initial Target Product Profile for potential future progenitor cell therapy application
  • Develop the basis for the submission of a CIRM TRAN1 grant application
Statement of Benefit to California:
In the United States, currently there are more than 17,900 new spinal cord injury (SCI) patients annually. Of particular concern in the California population are that SCI affects all races, with a higher incidence in Asians (28%), 24% in non-Hispanic blacks, 13.3% in Hispanics, and lower incidence in Caucasians and Native Americans. Our research aims to delineate the effectiveness of stem cell derived spinal interneurons to correct SCI deficits in order to develop better therapies.