Abstract
A vast spectrum of therapeutic interventions is based on stem cell technology, ranging from inert bio-materials to pluripotent, virally-modified cells. On the other hand, the scientific and regulatory community is concerned with the safety of stem cell-based products and therapies and the potential health risks associated with cell-based therapies. Scientific publications have revealed shared genetic similarities and cellular morphologic and phenotypic properties between stem cells obtained from embryonic and adult sources and cancer stem cells, thus creating considerable uncertainties about the long-term safety of stem cell-based products and therapies. Existing regulatory policy and guidelines are generally inadequate to address safety assessment and risk mitigation approaches to stem cell technology-derived products and therapies. Therefore, a need exists to develop core competencies within regulatory and standards-setting institutions to better understand the unique characteristics of stem cell-derived products and to perform safety assessments on a case-by-case basis, taking into account potential hazards and risk mitigation considerations. This article outlines an evidence-based risk mitigation framework that discusses two interrelated pathways in stem cell-based biotechnologies—stem cell-derived product development and stem cell-based therapies. The proposed conceptual framework integrates biohazard considerations for cell-based products with product and facility biosafety issues that are critical to research and product development activities. The stem cell-based clinical therapeutic pathway addresses the need for integrated Institutional Review Board and Institutional Biosafety Committee reviews as a way to develop core competencies to mitigate risks and ensure that institutional policy and safety considerations are adequately addressed in stem cell-based clinical therapy and product development activities.