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Survival guide to stem cell research and therapies: Part I

14. september 2015 at 8:00 | Veronika Valdova |  Medicine & Pharmacy
Stem cells offer tremendous promise for advancing human and animal health and medical science. Their potential is enormous: one day they may replace damaged tissues and even organs. Professionals and patients have high hopes for stem cell research because of their potential to treat conditions such as diabetes, multiple sclerosis, orthopedic degenerative diseases and injuries, spinal or brain injury, stroke, or myocardial infarction. But cell-based therapies are not the only use for stem cells. Other uses include basic research into cell division and differentiation and genetic and molecular controls, and testing new drugs. Safety of new drugs is tested on pluripotent lines; and cancer cells are used to test efficacy of potential new treatments [1].

Before use, stem cells must be manipulated so that they possess the necessary characteristics for successful differentiation, transplantation, and engraftment. This "minimal manipulation" has become the central point of stem cell regulation in the U.S. Stem cell therapies are regulated as biologics and are subject to premarket approval under the risk-based approach to approving cellular and tissue-based products. Treatments that are "minimally manipulated" are exceptions to this regulation and only have to comply with the current Good Tissue Practice[2].

Patient demand for new treatments in areas where effective cures are few and far between is enormous. It is difficult to estimate true numbers of patients seeking stem cell treatments in the U.S. and overseas. The most popular destinations include China, Mexico, Germany, the Dominican Republic, India, and Russia. Not all these countries offer equal protections to patients and standards in access to novel technologies. The global differences in regulation of research involving stem cells and novel therapies are vast.

Clinics all over the globe offer treatment of all kinds of conditions, from blindness, paralysis, multiple sclerosis, cerebral palsy, brain injuries and brain damage, and hundreds of others for sums ranging from $20,000 to 50,000. Offered treatments include allogeneic (from other humans) and xenogeneic stem cells (from animals and plants) as well as cells extracted from an individual, manipulated, and reinfused or re-implanted back into the same person (autologous stem cells). Despite uncertain effectiveness and possible severe side effects, stem cell treatments are gaining popularity. When patients are desperate, they may turn to experimental or unproven treatments:

"I may die anyway, but at least I died fighting. This is an experiment. What did I have to lose?" [3]

Some of the most egregious examples of unproven ad even dangerous stem cell treatments were discussed by Scott Pelley in CBS News investigation 60 Minutes in 2012 [4].

Development of U.S. policy on stem cell research
Source: Drug Development Primer: Stem cells, Arete-Zoe (2015)

In the U.S., the issue of stem cell research received nation-wide attention due to the utilization of surplus embryos from in-vitro fertilization (IVF) treatments as a major ethical concern. Presidential Executive Order 13505 from March 9, 2009 removed barriers to responsible scientific research involving human stem cells and changed the way the National Institute of Health (NIH) can support and conduct human stem cell research [5]. The 2009 Executive Order 13505 revoked previous Executive Order 13435 [6] of June 2007 and Presidential Statement of Aug 2001 [7] which prohibited federal funding for research on human embryonic stem cells (hESCs). In July 2009, NIH published Guidelines on Human Stem Cell Research on the donation of embryos for the derivation of hESCs, human induced pluripotent stem cells, and research involving human adult stem cells [8].

Minimally manipulated human cells and tissues and products derived from them are regulated solely under the Section 361 of the Public Health Service Act if they are intended for homologous use; if their manufacture does not involve the combination with another article, and if they do not have a systemic effect. Those that do not meet these criteria and do not qualify for exceptions are regulated as a drug, device, or biological product and have to be registered as such [9]. Whether this approach is sensible and appropriate and whether or not, and if so, how it should be changed is subject to fierce debate.

Registry of human stem cell and tissue establishments
In the U.S., human cell and tissue establishments are subject to registration. Registration details are publicly available in a database on FDA website.


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