iPS CELL THERAPY - Induced Pluripotent Stem Cells for Duchenne

Frequently Asked Questions (FAQs) about this research:
What stage is this research?
  • We had a Pre-IND meeting with the FDA in late March, and if everything goes well with our manufacturing and pre-clinical studies, we will be in the position for IND filling in the very near future..
Where is this research being done and who is funding this research?
  • This research is being done in the lab of Dr. Rita Perlingeiro at the University of Minnesota Center for Translational Medicine and the Molecular and Cellular Therapeutics Facility.  Seed funding has been kindly provided by the Greg Marzolf Jr Foundation. A grant submitted to the Department of Defense (DoD) has been recently recommended for funding.
What is the goal or purpose of this research?
  • Induced pluripotent stem cells (iPS) are adult cells that have been reprogrammed to an embryonic stem cell-like state. There has been tremendous excitement for the therapeutic potential of iPS cells in treating genetic diseases. Our current research builds on our successful proof-of-principle studies for Duchenne performed with mouse wild-type and dystrophic iPS cells as well as control (healthy) human iPS cells.  These studies demonstrate equivalent functional myogenic engraftment to that observed with their embryonic counterparts following their transplantation into dystrophic mice.
  • Our goal now is to apply this technology to clinical grade GMP-compliant iPS cells, and generate a cell product, iPS-derived myogenic progenitors, that can be delivered to muscular dystrophic patients. 
What steps need to be completed before moving into a clinical trial?
  • Optimization of methodology, characterization of cell product, scalability with GMP-compliant method, followed by safety and efficacy studies. Once these have been achieved, we will be ready to move into a clinical trial.
What is your best estimate for the length of time it will take to move this research into clinical trials?
  • 2-3 years (it depends largely on how much funding we have available to conduct these studies).
Where would a clinical trial take place?
  • University of Minnesota
Who would be eligible to participate in a clinical trial?
  • In the first phase, adults with confirmed diagnosis of DMD (> 18 years old).
Where can I learn more about this research?
(Updated 5/17/2017)