Caveat Emptor: New Guidelines are Issued to Protect Patients Against Bogus Stem Cell Therapies

Washington Post, December 3, 2008

As in any industry, an unmet need and the potential for economic gain often combine to produce two opposite results: on the one hand, such opportunity will attract legitimate experts who are authentically qualified to provide a beneficial service or product that meets a need, and, on the other hand, charlatans whose illigitimate services or products have no validity whatsoever will also be attracted to the field. Such has always been the case in most, if not all, economic sectors throughout history, especially in medical specializations, and such will probably always be the case throughout the future, given human nature and the tendency of history to repeat itself. Whether dealing with cars, jewelry or designer clothing, low quality reproductions of the most coveted styles abound and are often deliberately promoted as being something which they are not. It should come as no surprise, therefore, that the stem cell industry is no different. Fortunately, however, there is a simple "antidote" to such a danger: knowledge.

Since ancient times, free market economics have warned the buyer to beware, and fraudulent market activity is hardly a modern phenomenon. The very same market forces which allow for the possibility of deception on the part of the seller, however, also demand, and motivate, some level of intelligence and education on the part of the buyer. Predictably, therefore, deceptive products and services will often arise wherever legitimate opportunity and progress also exist, and history has repeatedly proven that this is usually not a question of "if" but rather a question of "when". In a population of educated and well-informed consumers, however, such deception will be shortlived, as knowledgeable people will be able to tell the difference between something of quality and value, as opposed to something that is worthless and perhaps even dangerous.

Unlike with cars, jewelry or designer clothing, the consumers of stem cell therapies are often patients with life-threatening disease or illness who are desperate for any treatment whatsoever. Consequently, scientists and governments alike are working to formulate official guidelines and regulatory laws that will protect the patient by ensuring, as much as is humanly possible, the legimacy of stem cell providers, and also by penalizing those who violate such regulation. Meanwhile, however, the stem cell field is still in its infancy, and the basic premises behind such regulation are not yet globally respected. Consequently, at the moment, anyone who wishes to peddle modern versions of snake oil while masquerading as a stem cell expert is free to do so, and those who actually do engage in such unethical and medically dangerous activity are tireless in their efforts to profit from the exploitation of consumers, especially with the ease of marketing their products and services over the internet.

According to Dr. Insoo Hyun, associate professor of bioethics at Case Western Reserve University School of Medicine in Cleveland, Ohio, and the lead author of a paper outlining the commercial guidelines of stem cell therapies, "Stem cell research is progressing so rapidly and has sparked a lot of interest in translational research including among patients in hope for therapies. At the same time, legitimate science is speeding ahead and getting to the point where there needs to be more of a roadmap to take the basic knowledge to clinical applications." Although such incidents have not yet made major news headlines, Dr. Hyun adds that it is "only a matter of time" before someone somewhere is physically harmed by bogus stem cell therapies.

According to Dr. Paul Sanberg, professor of neurosurgery and director of the University of South Florida Center for Aging and Brain Repair in Tampa, "We clearly need guidelines for around the world to make sure that appropriate research is done before clinical work is undertaken in patients. We see desperate patients all the time and want to make sure that any therapies they take come from responsible research groups." Similarly, Dr. Darwin Prockop, chair of Genomic Medicine and director of the Texas A&M Health Science Center College of Medicine Institute for Regenerative Medicine, adds, "There is tremendous confusion about the two types of stem cells, embryonic stem cells and adult progenitor stem cells. The difference is monumental, and needs to be clarified."

Although there do exist a number of clinics around the world which actually offer legitimate, scientifically based adult stem cell therapies, there also exist a number of clinics which do not offer anything of legitimacy, even though they purport to be legitimate. Accompanying the recent publication of stem cell guidelines is a commentary by Canadian researchers which contains an analysis of 19 websites that were found from a simple Google search, all of which advertize expensive stem cell therapies of dubious validity and safety. For example, a number of clinics in a number of countries such as China and the Ukraine claim to have treated thousands of patients for everything from Parkinson's disease and stroke to heart conditions, but without any scientific verification or corroboration of their claims. Indeed, a team of scientists from the University of Alberta were unable to find any substantiating evidence for any of the claims made by such clinics, nor were they even able to find any verification that real stem cells were actually used in the treatments, nor what types of stem cells might have been used, nor what the source of the cells might have been (human or nonhuman, for example, adult or fetal or embryonic, etc.). Additionally, nowhere were risks or contraindications mentioned on any of the websites for any of the clinics. Given the typical cost of such therapies, which averaged around $21,500, one might assume that prospective "customers" would be interested in obtaining prior verification of the safety and efficacy of such procedures, but apparently many patients who would otherwise have no treatment whatsoever are willing to take extraordinary risks, financially as well as medically, in exchange for even a false glimmer of hope, since even false hope is better than nothing, from the perspective of someone whose very life is already at risk.

According to Dr. Hyun, the newly formulated guidelines are meant to illuminate stem cell research and to guide researchers toward responsible and accountable practices, rather than to hinder or discourage their progress. Indeed, agencies such as the U.S. FDA (Food and Drug Administration) typically perform such roles, though not all countries have such regulatory governmental agencies. Furthermore, even in countries such as the U.S., where a powerful FDA has been governing medical research and clinical practices for years, specifically for the protection of human patients who are being treated with clinical therapies, nevertheless there is still the problem that such guidelines are outdated and do not apply to the stem cell field. The stem cell field is still young enough, and so radically different from all previous specializations of medical science, that the issues which are unique to stem cells have never before been fully addressed. As Dr. Hyun explains, "Most of the time, stem cell products are presenting entirely novel products that are unpredictable in humans. Unlike drugs, you can't just create a batch and put them on the shelf and expect they will be the same. We need uniform quality control and manufacturing. And if they're embryonic or pluripotent stem cells, they could form unwanted tissues or tumors. So, we have to be very careful about following up and monitoring patients."

Authored by a task force composed of stem cell specialists from 13 countries, the new guidelines address, among other topics, questions of ethical review, quality and safety, voluntary informed consent of participants in research projects, careful monitoring of volunteers, and caution in using stem cell therapies outside of a research context. Hopefully, the ethical principles which are at the very essence of such guidelines will be given serious attention and consideration by stem cell researchers throughout all countries of the world.

For those patients who are still awaiting stem cell cures for their diseases and illnesses, the field seems to be advancing much too slowly. As Dr. Hyun points out, however, not all progress is visible to the public. "For patients, it's not surprising that there are not direct applications," Dr. Hyun adds, "but what is often lost to the public is that so much knowledge has been gained from stem cell research. The advancements for patients are going to come sooner through these indirect routes, not direct cell-based therapy, but from the expansion of knowledge."

In addition to the need for ethical guidelines, there is another lesson to be learned from the increasing number of bogus stem cell therapies that are springing up around the world. Namely, the necessity and urgency of such guidelines also highlights the necessity and urgency for formal, official government approval of those adult stem cell therapies which have already been proven to be safe and efficacious, so that more clinics that offer such therapies will be allowed to open in their native countries, such as the United States, instead of having to locate themselves overseas in foreign countries where they are competing against the clinics that offer bogus therapies. In other words, an updated revision of the FDA approval process, so that it is directly relevant to stem cell therapies, would allow more adult stem cell therapies to be available throughout the U.S. to more patients with various diseases and injuries, who could benefit from such therapies but for whom such therapies have not yet received FDA approval. The current FDA approval process, which was designed years ago with the specific goal of testing safety and efficacy in pharmaceutical drugs, is in many ways neither relevant nor logical when applied to the testing of stem cell therapies. Such a topic is highly complex and could constitute an entirely separate publication unto itself. Suffice it to say that a swifter, more precise and more modernized FDA approval process which is specifically tailored to stem cell therapies is desperately and urgently needed in the United States, as is its equivalent in other countries.

Meanwhile, however, as in any market, consumers must arm themselves with the power of knowledge, which is their greatest defense. Especially where the quality of a product or service can make the difference between life and death, such as with stem cell therapies, it is all the more critically important that the buyer beware.