Identifying viable regulatory and innovation pathways for regenerative medicine: a case study of cultured red blood cells

N Biotechnol. 2015 Jan 25;32(1):180-90. doi: 10.1016/j.nbt.2014.07.008. Epub 2014 Aug 2.

Abstract

The creation of red blood cells for the blood transfusion markets represents a highly innovative application of regenerative medicine with a medium term (5-10 year) prospect for first clinical studies. This article describes a case study analysis of a project to derive red blood cells from human embryonic stem cells, including the systemic challenges arising from (i) the selection of appropriate and viable regulatory protocols and (ii) technological constraints related to stem cell manufacture and scale up to clinical Good Manufacturing Practice (GMP) standard. The method used for case study analysis (Analysis of Life Science Innovation Systems (ALSIS)) is also innovative, demonstrating a new approach to social and natural science collaboration to foresight product development pathways. Issues arising along the development pathway include cell manufacture and scale-up challenges, affected by regulatory demands emerging from the innovation ecosystem (preclinical testing and clinical trials). Our discussion reflects on the efforts being made by regulators to adapt the current pharmaceuticals-based regulatory model to an allogeneic regenerative medicine product and the broader lessons from this case study for successful innovation and translation of regenerative medicine therapies, including the role of methodological and regulatory innovation in future development in the field.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Biomedical Research
  • Cells, Cultured
  • Clinical Trials as Topic
  • Erythrocytes / cytology*
  • Humans
  • Inventions*
  • Regenerative Medicine / legislation & jurisprudence*