Recently passed legislation allows Texas clinics to bypass FDA approval for investigational stem cell treatments for patients with certain severe chronic diseases or terminal illnesses. The law alarmed ethicists who have been monitoring these practices.

“Despite years of research on the topic, the marketing of unproven stem cell therapies remains a significant and growing health policy problem,” says Timothy Caulfield, LLM, FRSC, FCAHS, research director at University of Alberta’s Health Law Institute in Canada.

Caulfield co-authored a recent paper advocating a call to action to address the marketing of unproven stem cell treatments.1 “The issue implicates tens of thousands of patients around the world. It hurts the legitimacy of good stem cell research,” says Caulfield.

The paper’s authors argue for a coordinated international response to regulate the situation. “Unless we develop a comprehensive strategy to deal with this issue, I think it is only going to get worse,” Caulfield says.

Ethicists believe the marketing of unproven treatments hurts legitimate stem cell research. “Tremendously exciting work is being done all over the world,” says Caulfield. “As the real therapies move closer to the clinic, it will become increasingly difficult to differentiate the real science from the bad.”

A recent paper argues that biotech companies should screen consumers to ensure that products and services are being used appropriately, and should educate employees about unproven stem cell interventions.2

“Currently, the U.S. has experienced a massive growth in the unproven stem cell therapy industry,” says Zubin Master, PhD, the paper’s lead author and a consultant at the Mayo Clinic’s Biomedical Ethics Research Program in Minneapolis.

Private clinics are selling unapproved, untested stem cell interventions directly to patients. “These have been shown to cause physical harms to patients. Also, many suffer financial hardship, and in some cases, emotional distress from a botched attempt,” says Master.

Some companies recently announced plans to supply private clinics selling unapproved stem cell interventions with stem cell reagents. Master and colleagues wanted to find out how informed these companies were about the unproven stem cell intervention marketplace. “We embarked on a study to identify whether they have policies to screen clients who may engage in selling unproven stem cell interventions, and whether they offer any education to their employees about the unproven stem cell intervention marketplace,” says Master. Some key findings include the following:

  • About one-quarter of the companies surveyed were aware of the International Society for Stem Cell Research or International Society for Cellular Therapy position statements on unapproved stem cell interventions.
  • About 20% of companies created policies to screen clients.
  • Only 7% provided any education about unproven stem cell interventions to their employees.
  • One respondent reported that clients sign contracts prior to the sale of the product, indicating that the end user will comply with the laws and policies in his or her country. If the client refuses to sign the contract, the company will not sell the product or offer the service.

“To curtail the direct-to-consumer sales of unproven stem cell therapies, everyone has to do their part,” says Master.

The researchers argue that legitimate industry also has a role to play. “It’s not just not the role of legislators and regulators to stop unethical business practices of providers,” says Master. “Scientists and the scientific industry also play a pivotal role.”

Douglas Sipp, another of the study’s authors, says, “If the government decides to leave the market for stem cell-based treatments unsupervised, this is almost certain to harm individual patients in the field of stem cell research, and the healthcare system in general.”

A number of high-profile incidents, including paralysis, blindness, and death, have been reported. “Evidence for the safety and efficacy of stem cells in human medicine is patchy and shallow in most cases,” notes Sipp, a research specialist at the Riken Center for Developmental Biology in Kobe, Japan.

Patients who pursue treatments they later discover to be harmful or fraudulent may have little legal recourse due to indemnifications for providers.

“Ethicists, along with concerned scientists and physicians, have been arguing against these dangerous laws for years — and will continue to do so, as long as they represent a threat to public health,” says Sipp.

Leigh Turner, PhD, associate professor at the University of Minnesota’s Center for Bioethics in Minneapolis, says of the Texas law, “In a way, what we have is a state carving out its own regulations that have the potential to undercut federal regulations.” This mirrors “right to try” laws being passed in a growing number of states.

“It wouldn’t be shocking if we see this in other states as well, with a lower regulatory bar in terms of bringing stem cell therapies to market,” says Turner. One reason is that businesses that open and market clinics have considerable resources to actively lobby for such legislation.

The argument to policymakers was that people were going to offshore clinics for the interventions, so they should be made available in the state. “But the whole debate was premised on a false assumption. The reality is, there are already 70 to 100 clinics in Texas that market stem cell treatments,” says Turner.

Turner says the real question is why clinics are marketing unproven treatments without adequate regulatory oversight: “State legislatures aren’t dealing with the problem in any meaningful way.”

In August 2017, the FDA announced it would strengthen regulatory oversight of clinics marketing stem cell treatments. The unanswered question, says Turner, is, “Are we going to see more enforcement action unfold, or is it going to be strong rhetoric without much action?”

As it stands now, clinics are making claims about therapies without credible scientific evidence. “All they have to do is put out a shingle on the internet and people know where to find them,” says Turner.

At some point, questionable claims become outright fraud. “We shouldn’t just be wringing our hands and standing there. People are at risk of having tens of thousands of dollars taken from them for nothing,” says Turner.

Turner says action must be taken before additional serious complications occur. “If these clinics are allowed to proliferate in the absence of regulatory oversight, we should expect more people being harmed,” he says.

REFERENCES

  1. Sipp D, Caulfield T, Kaye J, et al. Marketing of unproven stem cell-based interventions: A call to action. Sci Transl Med 2017; 9(397). pii: eaag0426. doi: 10.1126/scitranslmed.aag0426.
  2. Master Z, Fu W, Paciulli D, et al. Industry responsibilities in tackling direct-to-consumer marketing of unproven stem cell treatments. Clin Pharmacol Ther 2017; 102(2):177-179.

SOURCES

  • Timothy Caulfield, LLM, FRSC, FCAHS, Trudeau Fellow and Professor, Faculty of Law and School of Public Health/Research Director, Health Law Institute, University of Alberta, Canada. Phone: (780) 492-8358. Email: caulfield@ualberta.ca.
  • Zubin Master, PhD, Biomedical Ethics Research Program, Mayo Clinic, Rochester, MN. Phone: (507) 266-1105. Fax: (507) 538-0850. Email: master.zubin@mayo.edu.
  • Douglas Sipp, Research Specialist, Riken Center for Developmental Biology in Kobe, Japan. Email: sipp@cdb.riken.jp.
  • Leigh Turner, PhD, Associate Professor, Center for Bioethics, University of Minnesota, Minneapolis. Phone: (612) 626-4830. Fax: (612) 624-9108. Email: turne462@umn.edu.