Ann M. Parr, MN Neurosurgery, Minneapolis, USA

Autologous oligodendrocyte progenitor cell therapy in chronic spinal cord injury

Funded in: 2014, 2015, 2016

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Problem: After spinal cord injury, many cells and myelin sheaths are lost.

Target: Replacement strategies using oligodendrocyte precursor cells (OPC) derived from patient-specific induced pluripotent stems cells (iPS)

Goal: Improving the functional recovery and its correlation to myelination levels (in vivo MRI assay)

A possible treatment option for spinal cord injuries (SCI) is the transplantation of the patient’s own (autologous) oligodendrocyte progenitor cells (OPCs). Their clinical use will require functional, fully characterized OPCs to be reproducibly generated from patient-specific induced pluripotent stem (iPS) cells under conditions compliant for Good Manufacturing Practice (GMP). The use of patient-specific cells will prevent complications from long-term immune suppression and avoid ethical concerns about using cells derived from embryonic or fetal sources.

The aims of this proposal are:
i. Generate and characterize autologous OPCs from adult human individuals using iPS cells under GMP production conditions
ii. Demonstrate that these OPCs differentiate into mature oligodendrocytes after transplantation, and produce functional neurological recovery in the chronically injured rat spinal cord
iii. Utilize novel, non-invasive magnetic resonance imaging (MRI) methods to assess myelination in transplanted SCI as well as using MRI compatible cell labelling to track the transplanted cells in vivo.

At the University of Minnesota Stem Cell Institute, the group has already generated multiple iPS cell lines from human patients and is currently working to generate these cells under conditions that are GMP compliant. These human iPS cells will be further differentiated into OPCs.
In this project the ability of transplanted iPS cell-derived OPCs to improve functional recovery following SCI will be tested. The results will be included as preclinical data in support of an IND application for an FDA approved phase I clinical trial..