Spinal Cord Injury Fact Sheet

CIRM funds many projects seeking to better understand spinal cord injury and to translate those discoveries into new therapies.

Description

About 250,000 people in the U.S. live with spinal cord injuries. Half of those are quadriplegic, with the paralysis impacting all four limbs to some extent. For those individuals the lifetime cost of managing their condition is estimated to be between $2 million and $3 million.

Spinal cord injury became the first condition targeted in a human clinical trial using cells made from embryonic stem cells. That trial, begun by Geron in 2010 and based on the findings of a team CIRM currently funds, was later cancelled by Geron for financial reasons. By the time of the cancellation five patients around the country had been enrolled in the study, including two at Stanford, who entered the trial during a period when CIRM funded Geron. Those patients continue to be followed to learn as much as possible about this approach.

California’s stem cell agency retains many grants for research to move potential spinal cord injury therapies forward (the full list is below). Much of this work focuses on trying to determine which type of nerve cell is the best one to transplant, and deciding which type of stem cell is the best starting point for making those cells. Other research is trying to see if these transplanted cells become part of the existing nerve system, helping create new pathways that can transmit nerve signals to muscles. The researchers are also looking at ways to try and improve the ability of these transplanted cells to become part of the nerve system.

One obstacle that some teams are trying to overcome is the tendency of the scar at the site of injury to block the growth of these transplanted cells. One group is trying to overcome that by combining stem cells with synthetic scaffolds that can be placed at the site of injury, to help the cells bridge the scar and restore signals. In animal models this combination has resulted in an increase in mobility compared to stem cell grafts alone.

Clinical Stage Programs

Asterias Biotherapeutics (now called Lineage Cell Therapeutics Inc.)

The company uses cells derived from embryonic stem cells to heal the spinal cord at the site of injury. They mature the stem cells into cells called oligodendrocyte precursor cells that are injected at the site of injury where it is hoped they can repair the insulating layer, called myelin, that normally protects the nerves in the spinal cord. 


 
Progress and Promise toward a stem cell-based therapy for spinal cord injury

CIRM Grants Targeting Spinal Cord Injury

Researcher name Institution Grant Title Grant Type Award Amount
Eric Ahrens University of California, San Diego Molecular Imaging for Stem Cell Science and Clinical Application Research Leadership $5,680,474
Samantha Butler University of California, Los Angeles Assessing the mechanism by which the Bone Morphogenetic Proteins direct stem cell fate Basic Biology V $515,730
Francois Binette Lineage Cell Therapeutics, Inc. A Phase I/IIa Dose Escalation Safety Study of AST-OPC1 in Patients with Cervical Sensorimotor Complete Spinal Cord Injury Strategic Partnership III Track A $14,323,318
Sarah Heilshorn Stanford University Injectable Hydrogels for the Delivery, Maturation, and Engraftment of Clinically Relevant Numbers of Human Induced Pluripotent Stem Cell-Derived Neural Progenitors to the Central Nervous System Tools and Technologies III $1,347,767
Leif Havton University of California, Los Angeles Repair of Conus Medullaris/Cauda Equina Injury using Human ES Cell-Derived Motor Neurons Early Translational II $75,628
Bennett Novitch University of California, Los Angeles Molecular Characterization of hESC and hIPSC-Derived Spinal Motor Neurons Basic Biology I $1,228,278
Leif Havton University of California, Los Angeles Development of a Relevant Pre-Clinical Animal Model as a Tool to Evaluate Human Stem Cell-Derived Replacement Therapies for Motor Neuron Injuries and Degenerative Diseases Tools and Technologies III $1,308,711
Aileen Anderson University of California, Irvine Role of the microenvironment in human iPS and NSC fate and tumorigenesis Basic Biology II $1,256,194
David Schaffer University of California, Berkeley Scalable, Defined Production of Oligodendrocyte Precursor Cells to Treat Neural Disease and Injury Quest - Discovery Stage Research Projects $1,627,099
leif Havton University of California, Irvine Repair of Conus Medullaris/Cauda Equina Injury using Human ES Cell-Derived Motor Neurons Early Translational II $1,527,011
Mark Tuszynski University of California, San Diego Neural Stem Cell Relays for Severe Spinal Cord Injury Quest - Discovery Stage Research Projects $1,638,900
Binhai Zheng University of California, San Diego Genetic manipulation of human embryonic stem cells and its application in studying CNS development and repair SEED Grant $600,441
Aileen Anderson University of California, Irvine Generation and in vitro profiling of neural stem cell lines to predict in vivo efficacy for chronic cervical spinal cord injury. Quest - Discovery Stage Research Projects $1,575,613
Brian Cummings University of California, Irvine The Immunological Niche: Effect of immunosuppressant drugs on stem cell proliferation, gene expression, and differentiation in a model of spinal cord injury. SEED Grant $595,345
Mark Tuszynski University of California, San Diego Human Embryonic Stem Cell-Derived Neural Stem Cells for Severe Spinal Cord Injury (SCI) Therapeutic Translational Research Projects $6,235,897
Ziwei Huang Sanford Burnham Prebys Medical Discovery Institute New Chemokine-Derived Therapeutics Targeting Stem Cell Migration SEED Grant $708,000
John Brock University of California, San Diego Simplification of Excipient Solution for Implanting Candidate Human H9-scNSC Cell Line for Spinal Cord Injury Progression Award - Discovery Stage Research Projects $180,000
Hans Keirstead University of California, Irvine hESC-Derived Motor Neurons For the Treatment of Cervical Spinal Cord Injury Comprehensive Grant $2,158,445
Aileen Anderson University of California, Irvine A human neural stem cell therapeutic candidate for the treatment of chronic cervical spinal cord injury Therapeutic Translational Research Projects $4,950,024
Martin Marsala University of California, San Diego Spinal ischemic paraplegia: modulation by human embryonic stem cell implant. Comprehensive Grant $2,356,090
Sarah Heilshorn Stanford University Injectable, autologous iPSC-based therapy for spinal cord injury Quest - Discovery Stage Research Projects $789,000
Martin Marsala University of California, San Diego Induction of immune tolerance after spinal grafting of human ES-derived neural precursors Transplantation Immunology $1,387,800
Matthew Goodus BrainXell Therapeutics Autologous stem cell-derived interneuron cell therapy for spinal cord injury (SCI) Quest - Discovery Stage Research Projects $2,025,000
Jane Lebkowski Geron Corporation Evaluation of Safety and Preliminary Efficacy of Escalating Doses of GRNOPC1 in Subacute Spinal Cord Injury Targeted Clinical Development $0
Lyandysha Zholudeva Gladstone Institutes, J. David Excitatory spinal interneurons from human pluripotent stem cells to treat spinal cord injury Quest - Discovery Stage Research Projects $2,942,198
Arnold Kriegstein University of California, San Francisco Human ES cell-derived MGE inhibitory interneuron transplantation for spinal cord injury Early Translational III $1,623,251
Ioana Hone Lineage Cell Therapeutics, Inc. 1st Annual Spinal Cord Injury Investor Symposium (SCIIS) Conference II $50,000
Mark Tuszynski University of California, San Diego Functional Neural Relay Formation by Human Neural Stem Cell Grafting in Spinal Cord Injury Early Translational III $4,600,447
Total:
$63,306,661.00

CIRM Spinal Cord Injury Videos

Resources

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