Stem cells seem to be everywhere. We are constantly hearing discussions about the use of stem cells, regulatory issues for stem cells and the role for regenerative medicine. The conversations bridge multiple therapeutic areas including orthopedics, cosmetics, dermatology and even urology. One cannot go far without seeing an advertisement for a product containing stem cells or a physician or medical practice offering stem cell therapies. To date, the FDA has been very clear on its position on enriched stem cell products and therapies: NO. That is unless a company wants to file an IND, conduct a clinical trial and submit to the FDA as a biological product. Given the cost and delay of following that path, many stem cell therapies are instead being performed by clinicians as experimental, with patients paying cash for these procedures. This situation has not escaped the notice of the FDA. Many stem cell companies operating in this regulatory gray area have received warning letters, as in the cases of the cosmetic products and procedures highlighted in the below table.
| Company | Description of Warning Letter |
| Cell Vitals (Stem Cell Facial Cream) | Creams had components that could have drug-like activity and were not cleared by the FDA |
| Multiple skin care companies | Applications of plant stem cells have unevaluated potential risk |
| Multiple stem cell treatment clinics | Alteration of adipose-derived stem cells and the use of the cells in non-homologous application |
| IntelliCell Biosciences | Significant deviations from current good manufacturing practice |
| Crescent Health Center | Product labels misleading consumers; Product not recognized to be safe and efficacious |
| Hollywood Skincare International | Product cannot be marketed as safe without further approval from the FDA |
| Reviva Labs |
Numerous statements claiming to increase the volume of fatty tissue and reducing blemishes were deemed to be inaccurate |
As highlighted by these FDA concerns, stem cell products and procedures are not without risk. Nevertheless, patients are often willing to believe that their doctors are doing what is best for their patients in recommending a procedure, and doctors feel comfortable with what they perceive to be limited risk. This growth has led states to dig deeper. Could individual states force the FDA to reevaluate their position?
Several states have already passed laws allowing stem cell clinics to operate within the state, and currently about 60% of all US states are discussing similar legislation. All of these state actions are based on a belief that the FDA approval process for stem cell therapies is too long and costly, and an unwillingness to make patients wait for potentially life changing treatments. States are therefore allowing stem cell manufacturers and prescribing physicians to operate within the state with a greatly reduced exposure to regulatory risk. Although the FDA still has the ability to enforce federal laws, enforcement becomes more difficult after states have condoned the procedures. This circumstance is quite unique and is playing out in real time as the world watches.
What is the FDA’s stance? Cellular products such as stem cells must be registered with the FDA, and the claims companies can make about their products are tightly defined. These claims, in general, need to be backed up by clinical trial evidence. However, with the risk that clinical trials could unmask safety and efficacy issues, and the newly minted approval of some states to sell products and procedures without performing trials, there is little motivation for many companies to risk the FDA approval process. Many companies are instead opting to simply provide the therapies under the properly informed consent of their patients. Physicians are similarly motivated to continue with the status quo, marketing stem cell therapies as experimental treatments and charging whatever they want for the procedures. The regulatory confusion created by this unusual dynamic, and the resulting potential negative perception of the industry, have the potential to hurt cell therapy companies that want to go through the FDA processes and leave the FDA in an awkward position. Only time will tell if this will drive the FDA to try to prosecute non-compliant companies, or if it will push them to create more expedited pathways for cell therapies to be cleared.
At Locust Walk, we stay up to date on cell therapy technologies and have represented cell therapy companies in multiple capacities. We would be delighted to speak with you about assisting you or your company with achieving your goals. Please feel free to contact me at: stewart@locustwall.com.
