Disease Focus: Spinal Cord Injury
A Phase I/IIa Dose Escalation Safety Study of AST-OPC1 in Patients with Cervical Sensorimotor Complete Spinal Cord Injury
Up to 12,000 Americans suffer a spinal cord injury (SCI) each year. This condition leads to a high level of permanent disability and decreased life expectancy. Currently, there are no approved therapies for patients with SCI. Asterias Biotherapeutics uses cells derived from embryonic stem cells to heal the spinal cord at the site of injury. They […]
Evaluation of Safety and Preliminary Efficacy of Escalating Doses of GRNOPC1 in Subacute Spinal Cord Injury
Geron carried out a Phase 1 clinical trial to assess the safety and preliminary activity of escalating doses of human embryonic stem cell (hESC) derived oligodendrocyte progenitor cells for treatment of spinal cord injury. Five patients were treated, all safely and without any serious side effects, before the trial was halted by Geron for financial […]
Simplification of Excipient Solution for Implanting Candidate Human H9-scNSC Cell Line for Spinal Cord Injury
Excitatory spinal interneurons from human pluripotent stem cells to treat spinal cord injury
Research Objective The primary objective of this research is to test whether excitatory human V2a spinal interneurons engineered from PSCs can repair the damaged spinal cord and restore motor function. Impact Currently no existing therapies are capable of repairing the injured spinal cord. Our therapeutic cell candidate – human ‘V2a’ spinal interneurons – could address […]
Autologous stem cell-derived interneuron cell therapy for spinal cord injury (SCI)
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 […]
Injectable, autologous iPSC-based therapy for spinal cord injury
Research Objective We propose to develop and validate a therapy for spinal cord injuries in which human stem cell-derived neural cells is injected into the injured spinal cord using an injectable gel. Impact Our study will address the critical need for an SCI treatment that significantly improves the neurological recovery and hence quality of life […]
Generation and in vitro profiling of neural stem cell lines to predict in vivo efficacy for chronic cervical spinal cord injury.
Research Objective This project generates new cGMP compliant tissue educated human neural stem cell lines, paired with in vivo pre-clinical proof of concept testing, and development of a predictive in vitro profile. Impact Identification of new cell lines with in vivo efficacy testing to enable efficient translation to chronic cervical spinal cord injury, an area […]
Neural Stem Cell Relays for Severe Spinal Cord Injury
Research Objective We propose to utilize human neural stem cells to form neuronal relays across sites of severe SCI, restoring function across the site of spinal cord injury. Impact We will develop a specific type of neural stem cell that is best suited for repairing the injured spinal cord. Major Proposed Activities In Vitro Assessment […]
Scalable, Defined Production of Oligodendrocyte Precursor Cells to Treat Neural Disease and Injury
Research Objective The goal of this proposal is to develop an optimized, scalable process to manufacture high quality oligodendrocyte precursor cells (OPCs) from human pluripotent stem cells for treating human disease. Impact OPCs have therapeutic potential for spinal cord injury, restoration of cognitive function after cancer radiation therapy, inherited demyelinating disease, and potentially multiple sclerosis. […]
A human neural stem cell therapeutic candidate for the treatment of chronic cervical spinal cord injury
Translational Candidate The therapeutic candidate is a central nervous system tissue-derived GMP line developed under DISC2-10753 with an established GMP qualified seed bank. Area of Impact The target is chronic cervical spinal cord injury, which represents approximately 59% of clinical spinal cord injury cases. Mechanism of Action Integration of transplanted human neural stem cells is […]