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 NameInstitutionGrant TitleGrant TypeAward Amount
Leif HavtonUniversity of California, Los AngelesDevelopment of a Relevant Pre-Clinical Animal Model as a Tool to Evaluate Human Stem Cell-Derived Replacement Therapies for Motor Neuron Injuries and Degenerative DiseasesTools and Technologies III$1,308,711
Francois BinetteLineage Cell Therapeutics, Inc.A Phase I/IIa Dose Escalation Safety Study of AST-OPC1 in Patients with Cervical Sensorimotor Complete Spinal Cord InjuryStrategic Partnership III Track A$14,323,318
Dr Samantha J Butler Ph.D.University of California, Los AngelesAssessing the mechanism by which the Bone Morphogenetic Proteins direct stem cell fateBasic Biology V$515,730
Dr. Nobuko UchidaStemCells, Inc.Neural stem cell transplantation for chronic cervical spinal cord injuryDisease Team Therapy Development – Research$0
Mark TuszynskiUniversity of California, San DiegoFunctional Neural Relay Formation by Human Neural Stem Cell Grafting in Spinal Cord InjuryEarly Translational III$4,600,447
Dr. Arnold KriegsteinUniversity of California, San FranciscoHuman ES cell-derived MGE inhibitory interneuron transplantation for spinal cord injuryEarly Translational III$1,623,251
Dr. Jane Stephanie Lebkowski Dr.Geron CorporationEvaluation of Safety and Preliminary Efficacy of Escalating Doses of GRNOPC1 in Subacute Spinal Cord InjuryTargeted Clinical Development$0
Leif HavtonUniversity of California, Los AngelesRepair of Conus Medullaris/Cauda Equina Injury using Human ES Cell-Derived Motor NeuronsEarly Translational II$75,628
Leif A HavtonUniversity of California, IrvineRepair of Conus Medullaris/Cauda Equina Injury using Human ES Cell-Derived Motor NeuronsEarly Translational II$1,527,011
Dr. Eric T. AhrensUniversity of California, San DiegoMolecular Imaging for Stem Cell Science and Clinical ApplicationResearch Leadership$5,680,474
Dr. Zhigang HeUniversity of California, BerkeleyDeveloping a regeneration-based functional restoration treatment for spinal cord injuryResearch Leadership$0
Prof. Martin MarsalaUniversity of California, San DiegoInduction of immune tolerance after spinal grafting of human ES-derived neural precursorsTransplantation Immunology$1,387,800
Dr Hans S KeirsteadUniversity of California, IrvinehESC-Derived Motor Neurons For the Treatment of Cervical Spinal Cord InjuryComprehensive Grant$2,158,445
Prof. Martin MarsalaUniversity of California, San DiegoSpinal ischemic paraplegia: modulation by human embryonic stem cell implant.Comprehensive Grant$2,356,090
Dr. Benhai Zheng Dr.University of California, San DiegoGenetic manipulation of human embryonic stem cells and its application in studying CNS development and repairSEED Grant$600,441
Professor Ziwei HuangSanford Burnham Prebys Medical Discovery InstituteNew Chemokine-Derived Therapeutics Targeting Stem Cell MigrationSEED Grant$708,000
Dr. Brian J. CummingsUniversity of California, IrvineThe 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
Dr. Aileen J AndersonUniversity of California, IrvineRole of the microenvironment in human iPS and NSC fate and tumorigenesisBasic Biology II$1,256,194
Dr. Bennett G Novitch Ph.D.University of California, Los AngelesMolecular Characterization of hESC and hIPSC-Derived Spinal Motor NeuronsBasic Biology I$1,228,278
Armin BleschUniversity of California, San DiegoSimplification of Excipient Solution for Implanting Candidate Human H9-scNSC Cell Line for Spinal Cord InjuryProgression Award – Discovery Stage Research Projects$180,000
Lyandysha Viktorovna ZholudevaGladstone Institutes, J. DavidExcitatory spinal interneurons from human pluripotent stem cells to treat spinal cord injuryQuest – Discovery Stage Research Projects$2,942,198
Matthew GoodusBrainXell TherapeuticsAutologous stem cell-derived interneuron cell therapy for spinal cord injury (SCI)Quest – Discovery Stage Research Projects$2,025,000
Dr. Sarah C HeilshornStanford UniversityInjectable, autologous iPSC-based therapy for spinal cord injuryQuest – Discovery Stage Research Projects$789,000
Dr. Aileen J AndersonUniversity of California, IrvineGeneration 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
Mark TuszynskiUniversity of California, San DiegoNeural Stem Cell Relays for Severe Spinal Cord InjuryQuest – Discovery Stage Research Projects$1,638,900
David V. SchafferUniversity of California, BerkeleyScalable, Defined Production of Oligodendrocyte Precursor Cells to Treat Neural Disease and InjuryQuest – Discovery Stage Research Projects$1,627,099
Dr. Aileen J AndersonUniversity of California, IrvineA human neural stem cell therapeutic candidate for the treatment of chronic cervical spinal cord injuryTherapeutic Translational Research Projects$4,950,024
Mark TuszynskiUniversity of California, San DiegoHuman Embryonic Stem Cell-Derived Neural Stem Cells for Severe Spinal Cord Injury (SCI)Therapeutic Translational Research Projects$6,235,897
Ioana C HoneLineage Cell Therapeutics, Inc.1st Annual Spinal Cord Injury Investor Symposium (SCIIS)Conference II$50,000
Mark TuszynskiUniversity of California, San DiegoHuman Embryonic Stem Cell-Derived Neural Stem Cells for Severe Spinal Cord Injury (SCI)Late Stage Preclinical Projects$6,000,000
Sarah HeilshornStanford UniversityInjectable Hydrogels for the Delivery, Maturation, and Engraftment of Clinically Relevant Numbers of Human Induced Pluripotent Stem Cell-Derived Neural Progenitors to the Central Nervous SystemTools and Technologies III$1,347,767
Total:
$69,306,661.53

CIRM Spinal Cord Injury Videos

Spinal Cord Injury and Stem Cells: Jake Javier, Clinical Trial Participant

CURED: Stem Cell Clinical Trial Stories

Spinal Cord Injury and a CIRM-Funded Stem Cell-Based Trial

Spinal Cord Injury and Stem Cell-Based Trials: What’s the Latest?

World’s 1st Human Embryonic Stem Cell Trial for Spinal Cord Injury – Katie Sharify

Roman Reed: What all patient advocates at the World Stem Cell Summit should know

Bridges Trainee Meeting Keynote: Geron’s Embryonic Stem Cell Trial for Spinal Cord Injury

Progress and Promise in Spinal Cord Injury

Resources

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Stem Cell FAQ | Stem Cell Videos | What We Fund