The FDA heard public testimony on how to regulate the more than 500 stem cell clinics currently operating in the U.S. on the border line of medical ethics on September 12, 2016. The clinics are currently in a regulatory loophole with zero restriction on how untested stem therapies are advertised to consumers.
Professional athletes, pitchers in particular, make headlines when they go for experimental stem cell treatments over surgeries. But the industry also targets vulnerable patient populations like people with Alzheimer’s disease and children with autism. And the high cost of treatments, up to $100,000 are almost never covered by insurance, according to a study of these clinics by two academic stem cell researchers, Leigh Turner at the University of Minnesota School of Public Health and Paul Knoepfler of the University of California, Davis.
The FDA has approved only very limited stem cell treatment in the United States. Patients seeking other treatments often travel to Mexico and the Caribbean to receive the cells. Over the last decade, hundreds of U.S. clinics have popped up and operate with extremely limited oversight. The increase in clinics and their marketing prevalence prompted Turner and Knoepfler to catalog and publish their findings on the industry and its potentially fraudulent and unethical claims in the journal Cell Stem Cell.
They found 570 companies offering stem cell treatments around the U.S. with high geographic density in Beverley Hills, New York, Los Angeles and San Antonio, Texas. Most of these companies offer autologous stem cell transfer, meaning the cells come from the patient’s own supply. This is usually done by taking fat out of a person’s abdomen, concentrating the adipose stem cells and re-injecting. The efficacy of adipose-derived stem cells to treat anything is hotly contested, Knoepfler noted to the LA Times. Other clinics identified by the scientists advertise stem cells derived from amniotic fluid and umbilical cords without appropriate documentation of the cells sourcing. This is particularly controversial.
While some clinics specialize in plastic surgery or orthopedics, others advertise treatments for everything from neurodegernative diseases to sexual ailments, all under the same roof. From the Cell Stem Cell paper:
Other clinics take a much broader approach and list stem cell interventions for 30 or more diseases and injuries. Such businesses commonly market treatments for neurological disorders and other degenerative conditions, spinal cord injuries, immunological conditions, cardiac diseases, pulmonary disorders, ophthalmological diseases and injuries, and urological diseases as well as cosmetic indications. Many of these marketing claims raise significant ethical issues given the lack of peer-reviewed evidence that advertised stem cell interventions are safe and efficacious for the treatment of particular diseases. Such promotional claims also generate regulatory concerns due to apparent noncompliance with federal regulations.
The authors were most concerned that clinics were advertising for Alzheimer’s disease, Parkinson’s disease and autism, all conditions with vulnerable patient populations and conditions for which there is zero scientific consensus that such treatments work.
The clinics say they don’t fall under the FDA because they’re not using a pharmaceutical or medical device. And because they take cells from a patient and put them back into that same patient it does not count as a transplant. From Melinda Beck at the Wall Street Journal:
Clinic operators say they don’t need FDA approval because under the agency’s draft guidelines, stem cells aren’t considered drugs if they are the recipient’s own cells, they are not significantly altered and if they perform their original role in the target location.
But other critics say this argument is a dangerous loophole. By saying they don’t fall under the FDA’s purview, clinics can avoid the costly, long process of clinical trials necessary to prove the efficacy and safety of procedures. Instead, clinics can cash in by getting patients to pay for treatments that may or may not work and tracking potentially bad outcomes on a haphazard, voluntary basis. That will perpetuate a cycle of patients paying for treatments because there is no data about there efficacy.
On the other hand, it’s clear that patients want access to these treatments and feel they have a right to get it. From the Wall Street Journal:
SammyJo Wilkinson, a former dot-com executive, developed multiple sclerosis in 1995 and was confined to a wheelchair by 2011. She says her symptoms started to improve almost immediately after receiving a high-dose stem cell treatment at a Houston clinic in 2012. When the FDA blocked access to that form of therapy, Ms. Wilkinson went to Cancún, Mexico, for follow-ups. After a total of five treatments for $90,000, she says she has far less pain, can exercise and walk short distances with the help of a walker.
Wilkinson and other patient advocates have spurred potential legislation to secure access to stem cell treatments. The REGROW Act, now in committee, would fast-track some steam cell therapies, essentially letting them skip big costly phase III studies required by the established clinical trial protocol. From the bill:
Not later than 1 year after the date of enactment of this section, the Secretary shall establish a program to conditionally approve a cellular therapeutic product if the sponsor of such product demonstrates preliminary clinical evidence of safety, and a reasonable expectation of effectiveness, without initiation of phase III investigations.
But this argument isn’t a good one the editorial board of the journal Nature argues because most stem cell treatments haven’t gotten close to phase III. From the Nature editorial:
The claim that regulation is too harsh wrongly implies that the FDA is holding back therapies that work. Critics point to decades of preclinical and clinical work with stem cells and the pipelines of stem-cell treatments. With circular logic, they argue that, because the treatments have not been approved, there is something wrong with the approval system.
The FDA’s job is not to dictate who can get what treatments under what exceptional circumstances. Rather, it should define a regulatory process that keeps snake oil salesman from emptying the bank accounts of vulnerable people and simultaneously builds a framework to ensure safety and efficacy for treatment. From Nature:
The best way for the FDA to respond to the mood that has seeded the REGROW Act is to agree on a more efficient way to approve cell treatments. It is working to do so, but tensions are high… The FDA should strive to keep this debate on the proper topic — how to create a more efficient system that still scientifically evaluates whether treatments are safe and efficacious.
Meredith Knight is a frequent contributor to the human genetics section for Genetic Literacy Project and a freelance science and health writer in Austin, Texas. Follow her @meremereknight.