Conventional cell therapy has not delivered on its promise

Adult stem cells are regenerative populations of cells in developed organisms that are being pursued as therapeutics.  Bone-marrow derived mesenchymal stem cells (MSCs), also referred to as mesenchymal stromal cells, have shown significant therapeutic potential.

MSCs are an excellent candidate for cell therapy because:

  • Human MSCs are easily accessible from healthy donors.
  • The isolation of MSCs is straightforward and the cells can be expanded to clinical scales in a relatively short period of time.
  • Human trials of MSCs, thus far, have shown no adverse reactions to allogeneic (donor) MSCs.
  • MSCs secrete immunomodulatory, cytoprotective, and regenerative factors which have therapeutic properties.

MSCs have been shown to be effective in numerous preclinical models of disease including acute kidney injury, myocardial infarction, type I diabetes mellitus, and graft-versus-host disease, among many others.  Furthermore, the safety of MSC transplantation has been demonstrated in several clinical trials of cardiovascular, neurological, and immunological disease.  However, many of these trials have failed to meet primary endpoints for efficacy and thus the therapeutic promise of MSCs has not yet been realized.

The traditional routes of administration of MSCs – injection or intravenous infusion – have inherent limitations.  Studies have suggested that less than 1% of systemically administered MSCs persist for longer than a week following injection.  In addition, the maximum dose of MSCs that can be introduced safely into patients as a direct injection may not be sufficient to elicit a therapeutic response.  Our product is designed to overcome these limitations of conventional treatment.