There is strong interest among patient and medical communities alike in the potential of stem cell therapies to treat many diseases and medical conditions for which current treatment options are limited.
Many therapeutic developers are exploring the potential utility of stem cell therapies in a number of disease areas; however, very few have been granted approval by governmental regulatory agencies for commercial use.
At the same time, a growing number of “stem cell clinics” are taking advantage of the “hype” around stem cells and avoiding regulation by falsely marketing illegal and potentially harmful products to patients seeking cures for diseases, including the novel COVID-19 (coronavirus).
The criteria for determining if an HCT/P is not for “homologous use” or subject to more than “minimal manipulation” are defined in 21 CFR Part 1270.10 and FDA guidance. To ensure patient safety while realizing the significant promise of potentially curative cell therapies, ARM believes it is imperative that all HCT/Ps that fall outside these criteria adhere to appropriate regulations, such as Section 351 of the PHSA in the US, and undergo the appropriate, scientifically rigorous approval process overseen by duly empowered agencies, including the U.S. Food and Drug Administration (FDA), E.U. European Medicines Agency (EMA), Japanese Pharmaceuticals and Medical Devices Agency (PMDA), and others. Importantly, ARM requires its members to sign a Code of Conduct that compels them to adhere to these, and other, regulatory guidelines.
Public health is at risk when unscrupulous providers offer stem cell products that are unapproved, unproven and fail to adhere to established rules for good manufacturing practices. Many of these providers put patients at risk by falsely marketing the benefits of treatments, and often promoting the stem cells for conditions that are outside of their area of medical expertise.
ARM applauds international regulatory bodies for enforcement actions taken against unregulated stem cell clinics thus far, and believes that continued strong regulatory oversight of these clinics is imperative to protecting public health while continuing to foster medical innovation and development of safe, effective regenerative therapies.
Stem cell therapies have shown the potential to treat many diseases and medical conditions through the repair, restoration, replacement or regeneration of cells. There are stem cell therapies that have demonstrated clinical efficacy and been approved for commercial use by regulators; however, these are primarily limited to treatments for very rare diseases of the blood and immune system thus far.
Medicines regulators around the world require cell therapy product developers to satisfy a number of requirements before they may market a cell-based therapy for commercial use. These include: regulatory oversight of pre-clinical and clinical evaluation of the investigational product, adherence to appropriate pre- and/or post-market approval requirements, and demonstration that the product is being manufactured according to current good manufacturing practices (cGMP) to assure safety, purity and potency of a product before it reaches a patient.
Any claims made about the safety or effectiveness of a stem cell treatment should be proven through this scientifically rigorous regulatory process.
While FDA and others are applying a “risk-based approach” to enforcement of these regulatory requirements, the agency has stated it does not intend to exercise such enforcement discretion for those products that pose a potential significant safety concern to patients.
Many companies are developing cell therapies that adhere to the regulatory framework for regenerative therapies established by international regulators. In fact, several ARM member companies are conducting ongoing regulator-allowed clinical trials or preclinical studies for cell therapies intended to treat of a variety of chronic and acute conditions, including: chronic low back pain (DiscGenics and Mesoblast), delayed union fractures (Bone Therapeutics), graft versus host disease (Mesoblast), ischemic stroke (Athersys), Parkinson’s disease (BlueRock Therapeutics), type 1 diabetes (ViaCyte), and urinary incontinence (CookMyosite).
However, there are more than 700 stem cell clinics operating within the United States alone1, and many more across Latin America, Europe, Asia, and beyond, often selling unproven, unsafe and potentially harmful products to patients with serious illnesses. These predatory clinics are advertising unproven therapies as “cures”—for a price—and are preying on patients desperate to find a treatment for their particular condition because conventional medicine has failed to provide a solution. This results in a dangerous trend toward stem cell tourism where the price tags for patients, the risks associated with these unproven treatments, and the profits to the clinic are all significant.
