13(Public Health Services Act, Section 262(i) 2011 ). Similar to vaccines, stem cells
can be therapeutically used to reconstitute or strengthen a patient’s immune system,
and stem cells derived from umbilical cord blood also qualify as blood products
(Munzer 2012 ). To use stem cells therapeutically also requires that cells be deliv-
ered to specifi c locations in the body which may require the use of a device to aid
the cells in this regard, which yields another form of combination product (FDA
2010 ). It is clear that the categorization of stem cells is challenging and further
complicated by external factors such as cryopreservation which raises concerns
about their stability and requires safeguards pertaining to pre-freeze and post-thaw
preservation (Center for Biologics Evaluation and Research (CBER) 2008 ). Stem
cells can support the growth of pathogens and are unable to be sterilized. When
delivered to sensitive areas such as the central nervous system, this may further
complicate their categorization (Barinaga 2000 ).
In December 2014, the Food and Drug Administration (FDA)—the American
regulatory authority—released two draft guidance documents (FDA 2014a , b ),
describing its view on the preparation of MSCs from fat and their use in patients. In
essence, these draft guidelines recommend that MSCs are to be regarded as biologi-
cal drugs, meaning that the provider/manufacturer will have to prove benefi t in the
clinical trial setting before they will be reviewed and considered for marketing
approval by the FDA. If this recommendation informs the FDA’s decisions in this
regard, no clinic in the USA will be authorized to offer unproven MSC products. It
is anticipated that regulatory authorities in other major markets will follow suit,
particularly the European Union.
1.3.2 Minimal Manipulation Requirements
Many biotechnology companies try to fast-track stem cell-based treatments to mar-
ket without the prescribed regulatory approval or registration by arguing that their
stem cell-based products are minimally manipulated and do not fall within the ambit
of such regulations. A similar argument was proffered in the Regenexx case in
which the classifi cation of stem cell therapy as biological medicine with regard to
autologous stem cell therapy, where a patient’s own stem cells are administered
back to the same patient after having been processed, cultured, mixed with other
therapeutic substances, stored, or even cryopreserved, was challenged (Jordaan
2012 ). In this case the FDA claimed that the autologous stem cell-based substance
produced using the Regenexx procedure not only qualifi es as a “biological product,”
but the offered cell treatments were more than minimally manipulated and therefore
fall within the regulatory ambit of the FDA and need FDA approval and registration
(United States of America v. Regenerative Sciences 2014 ). The FDA subsequently
ordered its developers, Regenerative Sciences, to stop offering their unapproved
biological drug product.
1 Stem Cell Therapy: Accepted Therapies, Managing the Hope of Society...