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Last September , the FDA held public hearings to address industry and stakeholder concerns regarding four draft guidance documents detailing the FDA ’ s interpretation of stem cell regulations .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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