Stem Cell Therapies: Opportunities for Ensuring the Quality and Safety of Clinical Offerings: Summary of a Joint Workshop by the Institute of Medicine, the National Academy of Sciences, and the International Society for Stem Cell Research (2014)

E

Glossary

Allogeneic: refers to cells or tissues from different individuals of the same species, but that are genetically different from each other.

Autologous: refers to cells or tissues derived from the same individual and are therefore genetically identical.

Clinical trial: any investigation in human subjects intended to discover or verify the clinical, pharmacological, and/or other pharmacodynamic effects of one or more investigational medicinal product(s), and/or to identify any adverse reactions to one or more investigational medicinal product(s), and/or to study absorption, distribution, metabolism, and excretion of one or more investigational medicinal product(s) with the object of ascertaining its (their) safety and/or efficacy.¹

Efficacy (or therapeutic efficacy): the measure of the performance of a treatment for a given indication.

In vitro fertilization: combining eggs and sperm outside of the body with the goal of the sperm fertilizing (entering) the egg. The fertilized eggs (embryos) are then transferred to the uterus for implantation.

__________________

¹International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use, Guidance for Industry E6 Good Clinical Practice: Consolidated Guidance Guideline for Good Clinical Practice. See http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM073122.pdf (accessed April 8, 2014).

Institutional review board (IRB): a committee that reviews, approves, and monitors biomedical research involving human subjects for the purpose of protecting the rights and welfare of participants.²

Medical tourism: the act of traveling across borders to seek medical treatment.

Mesenchymal stem cells: multipotent cells that can differentiate into bone (osteocytes), cartilage (chondrocytes), fat (adipocytes), and possibly other cells.³

Minimally manipulated: the processing of cells in a way that does not alter their relevant biological characteristics.⁴

Nutraceutical: an ingestible foodstuff that is purported to impart health or medicinal benefit.

Pluripotency: the cellular characteristic of being able to differentiate into all specialized cell types.

Regenerative medicine: an area of biomedical research focused on developing treatments for the repair, replacement, or regeneration of damaged cells, tissues, or organs.

Regulatory science: the development and use of new tools, standards and approaches to more efficiently develop products and to more effectively evaluate product safety, efficacy, and quality.⁵

Stem cell: a general term for cells that have the ability to self-renew and differentiate into a variety of mature specialized cell types.

__________________

²FDA Regulatory Information. See http://www.fda.gov/regulatoryinformation/guidances/ucm126420.htm (accessed April 8, 2014).

³Also known as multipotent stromal cells, there is controversy over whether these are actually stem cells, including whether they can perpetually self-renew and differentiate.

⁴FDA CFR—Code of Federal Regulations Title 21 revised 2013. See http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/cfrsearch.cfm?fr=1271.3 (accessed April 7, 2014).

⁵FDA News and Events. See http://www.fda/gov/NewsEvents/Newsroom/PressAnnouncements/ucm201706.htm (accessed April 8, 2014).

Stem cell tourism: a type of medical tourism whereby a patient travels across borders to seek stem cell therapies to treat or cure disease.

This page intentionally left blank.