THE BASICS OF STEM CELL THERAPY REGULATION
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Some call for strict regulation through bodies such as FDA, while others argue strict regulation will only curtail the benefits stem cell therapies can impart.
In recent years, clinics offering regenerative therapies( sometimes referred to as stem cell clinics) have proliferated in the United States. Now that the treatments are being offered much more widely, the Food and Drug Administration( FDA) has become increasingly aggressive in its regulation of stem cell therapies, with regulations that generally call for the same rigorous testing procedures currently required for drugs and devices.
Last September, the FDA held public hearings to address industry and stakeholder concerns regarding four draft guidance documents detailing the FDA’ s interpretation of stem cell regulations. Doctors, business owners, academics, and patients spoke at the two-day event and weighed in on what they think needs to change.
Currently, how a clinic or other provider of regenerative medicine therapies is regulated depends on what services that establishment offers. Some may be exempted entirely, some may be subject to the infectious disease controls, and others may be required to seek FDA approval.
To understand what services an establishment offers, three concepts are important. First, stem cells are either autologous, meaning that they come from the same individual, or allogenic,
meaning that they come from a different individual. Second, stem cells may be used for a homologous purpose or a nonhomologous purpose. A homologous use or purpose is one where the stem cells are used for the same type or purpose as the origin of that particular stem cell. Thus, a homologous use for a stem cell obtained from the bone marrow would be for a blood or hematological condition. A nonhomologous use would be for any other use or purpose. Using umbilical cord blood stem cells to treat a disease such as multiple sclerosis or ALS would be a nonhomologous use. Third, stem cells may be“ more than minimally manipulated,” which means the cells have been changed or altered in a significant way. With these three concepts in mind, let’ s turn to the regulations.
FDA regulations establish two broad categories for what it terms human cells, tissues, and cellular and tissue-based products( HCT / Ps). HCT / Ps that fall under a part of the rule known as section 361 are self-derived( autologous), minimally manipulated, and used in a homologous manner. They do not need an Investigational New Drug( IND) approval. Other HCT / Ps are governed by section 351. Section 351 products are those that are more than minimally manipulated or ones that are used in a nonhomologous manner. These products are considered indistinguishable from drugs and must undergo a rigorous regulatory process, including an IND, preclinical safety studies, clinical trials, and marketing approval before being given to patients. This is a very big deal in terms of time, expense, and volume of paperwork.
Issues remain regarding how best to regulate the use of stem cell therapies. Some call for strict regulation through bodies such as the FDA, while others argue strict regulation will only curtail the benefits stem cell therapies can impart. But, regulations that are too lenient, it is argued, will only harm the patient seeking the therapy, as a solid evidence base will not yet have been compiled for the therapy. Finding a regulatory middle ground for unproven stem cell therapies is going to be a challenge, and undoubtedly, much more discussion will ensue as this field moves forward.
Legal counsel representing regenerative medicine companies should continue to monitor these rules for any changes that affect or improve stem cell therapy opportunities.
Author: Thomas( T. J.) Ferrante- Foley & Lardner, LLP
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