Safety of Autologous Human Schwann Cells (ahSC) in Subjects With Subacute SCI
Condition: Spinal Cord Injury; Paraplegia
MeSH terms: Spinal Cord Injuries; Paraplegia
Study type: Interventional
Phase: Phase 1

Summary: 
      The purpose of this study is to assess the safety of autologous human Schwann cells (ahSC)
      transplantation in subjects with subacute SCI.

      For humans with subacute SCI, we hypothesize that axons might show improved function if
      myelin repair is induced with the implantation of ahSC. In addition spinal cord cavitation
      may be reduced, and neural sprouting and plasticity may be enhanced via neurotrophic effects.
    

Detailed description: 
      Schwann cells are excellent candidates for transplantation into humans with SCI. Large
      numbers of ahSC can be derived for autologous implantation after a minor surgery for
      peripheral nerve harvesting, and purification and expansion of the cells in culture.
      Autologous cells offer important safety advantages that include no need for immune
      suppression, minimal risk of disease transfer, and a low risk of tumorigenicity.

      Since 1990, scientists at the Miami Project to Cure Paralysis have generated extensive
      preclinical data suggesting Schwann cell transplantations are successful in rodents with SCI.
      The most recent work has focused on contusive injury models that are relevant to human
      injury. They have also been largely responsible for developing an efficient method for
      procuring large, essentially pure populations of human Schwann cells from adult peripheral
      nerve.

      The rationale for implantation of ahSC in people with acute SCI is based on the evidence that
      Schwann cells are neuroprotective and are capable of myelinating axons. Using mitogen
      expanded human Schwann cells in SCID mice and athymic female nude rats demonstrated that
      human Schwann cells can survive and are capable of enhancing axonal regeneration and forming
      myelin after transplantation in animals with sciatic nerve transection or thoracic spinal
      cord transection. The proposed clinical trial will advance knowledge about the safety and
      feasibility of a cell-based treatment strategy for human SCI.

      This Phase 1 clinical trial will employ an open label, unblinded, nonrandomized and
      non-placebo controlled dose-escalation design to evaluate the safety of transplantation of
      ahSC transplantation in subjects with subacute SCI.

      A sural nerve harvest will occur within 30 days post-injury. Standard-of-care medical
      treatment and rehabilitation will proceed while the cells are being processed in a cGMP
      facility. No later than 72 days post-injury, the ahSC product will be administered via a
      single injection into the cavity of the spinal cord lesion.

      Safety and efficacy assessments will be performed at week 1 and 2 post-transplantation and 2,
      6, and 12 months post-transplantation.
    

Intervention name(s): Autologous Human Schwann Cells
Intervention type(s): Biological
Intervention description(s): Schwann cells harvested from the sural nerve of the participant will be autologously transplanted into the epicenter of the participant's spinal cord injury.
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: paraplegia; cell therapy; Schwann cells; spinal cord injury