UPDATE ON “STEM CELLS” and AMNIOGENIC CELL THERAPY PRODUCTS
Recently, there has been a rapid increase of marketing by medical clinics/providers offering “stem cell” therapy to “cure or treat” arthritis and other musculoskeletal disorders. Many of these “remedies” are being offered by providers who have no training, knowledge or expertise in the musculoskeletal system at all and are not familiar with the biology, physiology, anatomy nor appropriate validated proven treatments. Unsuspecting patients have shelled out hundreds and thousands of dollars for unproven treatments. At AZISKS, Dr. Bailie believes in the importance of staying current in the medical field. He is involved in clinical trials and research continuing to advance medical care in the US for Orthopedic conditions. He has co-developed 2 shoulder replacements and has participated as a Principal Investigator in FDA IDE Studies. He frequently shares his outcomes while lecturing around the world, and publishes, in peer reviewed journals, textbooks, and other scientific literature.
As “regenerative medicine” is an area of intense study and interest, AZISKS endeavors to educate all of our patients and the community regarding what is actually legitimate and what may be inappropriate use of treatments for the purposes of financial gain. The information below is from direct communication Dr. Bailie has had with the FDA regarding AMNIOGENIC cell treatments (via injectable material) and “stem cells”. For further data, please see below for FDA contact information or you may reach out to Dr. Bailie directly via email.
FROM Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER).
One of seven centers within FDA, CBER is responsible for the regulation of many biologically-derived products including blood intended for transfusion, blood components and derivatives, vaccines, allergenic extracts, tissues, and cell and gene therapy products.
Cellular therapies, including regenerative medicine products, are an incredibly important class of medical products. In theory, stem cells may advance the treatment of many other diseases or conditions; however, at this time, the value of stem cells as a treatment for most conditions is largely unproven and more information is needed about their potential benefits.
Currently, there are no injectable amniotic membrane products approved by the FDA for use in the United States. At this time, the only stem cell-based products approved by FDA for use in the U.S. are hematopoietic progenitor cells cord blood (blood forming stem cells) that are isolated from placental or umbilical cord blood. Several different sponsors hold licenses for HPC, Cord Blood. These products are approved for a limited number of uses in patients with disorders affecting the hematopoietic (blood forming) system that are inherited, acquired, or result from treatment that destroys the existing stem cells. They are not approved for injection into joints or other musculoskeletal diseases.
A list of approved cellular and gene therapy products can be found here: https://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/default.htm
As you may be aware, amniotic fluid and other cellular and tissue products are regulated as Human Cells, Tissue, and Cellular and Tissue-based Products (HCT/Ps). The regulations in Title 21 Code of Federal Regulation (CFR) Section 1271.10(a) identify the criteria for regulation solely under Section 361 of the Public Health Service (PHS) Act. If all the criteria in 21 CFR Section 1271.10 are met, then no pre-market review (application to FDA) is required. To satisfy these criteria, an HCT/P must be: No more than minimally manipulated (relates to the nature and degree of processing); intended for homologous use only (the product performs the same basic function in the donor as in the recipient); not combined with another article (with some limited exceptions); and the HCT/P does not have a systemic effect and is not dependent on the metabolic activity of living cells for its primary function, or if it does, the HCT/P is intended for autologous use or use by a first- or second-degree blood relative.
HCT/Ps that do not meet all of the criteria in 21 CFR Section 1271.10 are also regulated under Section 351 of the PHS Act and/or the Federal Food, Drug and Cosmetic Act as drugs, devices and/or biological products. The investigational use of 351 products requires the submission of an investigational new drug (IND) application or an investigational device exemption (IDE). INDs and IDEs allow the investigational product to be used in a clinical study in order to collect safety and effectiveness data required to support the approval of a biologics license application (BLA), or a premarket approval (PMA) application in the case of a device.
Further background about this comprehensive policy framework for the development and oversight of regenerative medicine products, including novel cellular therapies can be found at
https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm585345.htm
https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm585342.htm
The FDA is concerned that the hope patients have for treatments not yet proven to be safe and effective may leave them vulnerable to unscrupulous providers of stem cell treatments that are illegal and potentially harmful. The FDA encourages consumers to learn all they can about the regulations covering any stem cell-based product, to educate and advise consumers about unproven and potentially unsafe stem cell therapies. Individuals considering stem cell therapy are urged to ask questions to ensure that the necessary FDA approval has been obtained or that they will be part of an FDA-regulated clinical study conducted under an active IND on file with the FDA.
Additionally, consumers are encouraged to contact the FDA and the appropriate state authorities in their jurisdictions to report any potentially illegal or harmful activity related to stem-cell based products.
For additional information please refer to the FDA website for our warning to consumers regarding stem cell therapies.
The regulation of a tissue-derived product and the determination of whether it needs FDA review and approval before marketing, depends on careful evaluation of all the criteria detailed above. For products that do not meet all of the criteria, a BLA or PMA would need to be submitted to FDA for review and approval before commercial marketing occurs. The FDA expects anyone involved with recovering, processing, or otherwise manufacturing HCT/Ps to familiarize themselves with the regulations; they are responsible for complying with the regulations that apply to their products. FDA has created the Tissue Reference Group (TRG) to respond to questions from sponsors, investigators and manufacturers about how the 1271.10(a) criteria described above apply to specific HCT/Ps. Additional information about the TRG, as well as previous determinations, can be found at the following link:http://www.fda.gov/biologicsbloodvaccines/tissuetissueproducts/regulationoftissues/ucm152857.htm.
The FDA is committed to ensuring that patients have access to safe and effective regenerative medicine products, within the framework of FDA regulation. The FDA has adopted a risk-based and science-based approach that builds upon existing regulations to support innovative product development while clarifying the FDA’s authorities and enforcement priorities. This risk-based approach allows product developers time to engage with the FDA, to determine if they need to submit a marketing authorization application and, if so, submit their application to the FDA for approval.
To reach the FDA for more information please contact:
ocod@fda.hhs.gov or 1-800-835-4709.
