Unproven Stem Cell Therapies: Experts Discuss How To Protect Yourself From This Global Health Issue

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Off the side of the highway, between billboards for amusement parks and car dealerships, you may have encountered a new type of advertisement: “Cure your arthritis!” or “Get rid of your pain!” these billboards boast — “It’s all possible with the power of your own stem cells!”

While many promising stem cell therapies are in development for heart disease, macular degeneration, Parkinson’s disease, and more, ‘bad actor’ clinics worldwide are exploiting the excitement around stem cells to sell expensive, unproven therapies to vulnerable patients with limited options. 

“In recent years, we have seen a huge proliferation of unproven stem cell therapies offered around the world and right here in the United States,” remarked NYSCF CEO Susan L. Solomon, JD. “There are known and proven stem cell therapies on the market today, most significantly bone marrow transplants that have been transformational for many cancer patients; however, the vast majority of stem cell treatments that are marketed for a huge breadth of devastating diseases are not only unproven – which means there’s no data to show that they work – but they also can be harmful to patients.”

We recently convened experts Peter Marks, MD, PhD (U.S. Food and Drug Administration), Timothy Caulfield, LLM, FRSC, FCAHS (University of Alberta), Valentina Fossati, PhD (The NYSCF Research Institute), and Jeffrey Kahn, PhD, MPH (Johns Hopkins Berman Institute of Bioethics) to explore the science behind legitimate stem cell therapies and what these unproven stem cell clinics are offering patients.

What are stem cells, and how do they work?

The two central features of stem cells are their ability to self-renew (create more of themselves) and become tissue or organ-specific cells with specialized functions.

“Our body has a remarkable regenerative capacity,” explained Dr. Fossati. “For example, we can donate blood regularly because our blood is constantly replenished by a reservoir of stem cells that live in the bone marrow.”

Dr. Fossati’s research uses stem cells to study diseases of the brain.

“In the past 15 years, scientists have learned how to take skin or blood samples and make what we call ‘induced pluripotent stem cells’ in a dish,” she said. “For my work, I turn them into brain cells to study diseases like multiple sclerosis and Alzheimer’s, but they can be turned into virtually any type of cell in the body.”

Stem-cell-derived astrocyte, a support cell in the brain. Image credit: Lili Barbar, NYSCF

Stem cells that exist in specific parts of the body and create a certain cell type are called adult stem cells. These are the stem cells used, for example, in lifesaving bone marrow transplants. However, some types of adult stem cells are being used in unproven therapies that can be harmful to patients: causing everything from tumors to blindness.

“There is another population of adult stem cells called mesenchymal stem cells that are found in bone marrow, as well as in adipose tissue (fat), cord blood, and the placenta. These are the cells that are most commonly used and sold as an unlicensed product by some clinics,” noted Dr. Fossati.

How do predatory clinics bypass FDA regulations?

One reason the FDA has traditionally found it difficult to regulate these clinics is because it is unclear if the clinics fall within FDA oversight. 

“The FDA looks to regulate things in the least burdensome way we can while also keeping products safe,” explained Dr. Marks. “So for certain stem cell products, like when one is taking bone marrow from one individual and giving it to another as part of a stem cell transplant, there’s a very low level of regulation there. We don’t get involved with that. And because of those types of exceptions, there’s sometimes some confusion.”

Unproven stem cell treatments typically follow the same pattern: cells are removed from a patient and then injected back into a different part of the patient’s body. But they are still the patient’s own cells, and since they fall under a category classifying them as “minimally manipulated,” the FDA is limited in how much it can intervene. 

“There is a set of regulations called tissue regulations which categorize certain products as minimally manipulated – meaning that they are used in the same manner in the recipient as they are in the donor,” said Dr. Marks. “They aren’t mixed with another product, and they don’t require the same demonstrations of safety and efficacy as manipulated stem cells might. So [stem cell clinics] will claim these exemptions and circumvent all this to say it’s already approved and they can sell it, because identifying which category you fall into is a self-determination.”

“It isn’t like the FDA said there’s no reason to regulate these things, it’s that it just doesn’t fit the typical model,” added Dr. Khan. “That’s the story of reproductive technologies [such as IVF] too, which doesn’t see the kind of FDA regulation that other products do, and there’s been a long history of reproductive technology tourism. And so it’s easy [for predatory stem cell clinics] to say, well, we know how this works in this other area that’s lightly regulated, so it’s just an easy application of that history.”

How have predatory stem cell clinics evolved, and how do they draw people in?

“It is a fascinating evolution – I started studying stem cell tourism around 2005, and when it began, it was largely people leaving the United States and going to particular jurisdictions like China, Ukraine, Russia, Mexico, or the Caribbean,” remarked Mr. Caulfield. “These clinics were leveraging the legitimate excitement around stem cells to sell entirely unproven therapies.”

Now, the need to go abroad has diminished due to the boom of clinics popping up around the United States.

“Over the last 10 years or so, we’ve seen an incredible growth of domestic clinics,” he continued. “There was a recent study from my colleague Leigh Turner that identified nearly 3,000 clinics in the United States.”

Mr. Caulfield noted that predatory clinics employ an arsenal of marketing techniques that circumvent data-driven arguments.

“We see very creative marketing around this stuff to get around the FDA,” said Mr. Caulfield. “Clinics will use a lot of testimonials rather than actual data, which is a big red flag, or they’ll hold seminars and have people come hear about stem cell therapy.”

Unproven stem cell treatments have even reached some of America’s biggest sports stars, offering a sense of false legitimacy.

“There are athletes who will talk about getting a stem cell treatment, and that’s covered in sports media,” explained Mr. Caulfield. 

“If your favorite running back gets a stem cell treatment, then it makes it seem more legitimate. I call this ‘scienceploitation,’ where someone takes real, exciting science and uses it to market bunk.”

“Another really good example is crowdfunding,” added Mr. Caulfield. “Another colleague of mine, Jeremy Snyder, has done some really interesting studies looking at crowdfunding for unproven stem cell therapies. This is a very powerful form of marketing because it’s patients speaking to patients.”

What does ‘Right to Try’ have to do with unproven treatments?

A policy called ‘Right to Try’ can muddy the waters when it comes to whether therapies are safe to try, and can create a false perception that the FDA is creating barriers to treatments.

“[Right to Try] was something that was put forward a few years ago stating that people without other possibilities for therapy should have the right to try whatever is out there,” explained Dr. Marks. “What was eventually agreed upon as part of Right to Try is that for something to be eligible, meaning that it can be used without informed consent, not on a clinical trial, and not under any kind of FDA oversight, it does need to be under IND investigation, have a new drug application with FDA, and it needs to be past phase one. In other words, it has to be shown to be generally safe. And we grant roughly 99% of these expanded access requests.”

“People understand things like ‘compassionate use’ and ‘expanded access’ and early access to drugs in clinical trials as all living under Right to Try as a general term, but Right to Try has a very specific meaning,” added Dr. Khan. “This plays into this problem of potential exploitation.”

“This plays to the myth that FDA is withholding efficacious treatments from the public and can be used to legitimize unproven therapies,” remarked Mr. Caulfield.

How can you strike the right balance between caution and optimism when it comes to investigational therapies?

“Usually despite public perception, when something is in a clinical trial, it usually doesn’t make it to clinical application,” noted Mr. Caulfield. “The number of therapies that pass clinical trials hovers around 10%. And if it does make it to the clinic, it is usually less efficacious than promised.”

“As a hematologist oncologist who treated a lot of leukemia patients – many with advanced leukemia – I’m very aware of what people think when they’re getting an investigational therapy. People have an expectation and that expectation may not match up with reality,” added Dr. Marks.

“Part of the challenge here is that heart-wrenching stories often are used to justify broad policy,” noted Dr. Kahn. “So for example, if there’s a very sick child for whom the only therapy that could possibly work is something that’s in a trial and the family wants access to it, and they don’t survive. And then it’s pointed to as a problem, implying that the process is too slow and it’s denying lifesaving care to people who need it. It’s like they say in the legal field: really dramatic cases make for bad law.”

The good news is that there are effective stem cell-based therapies entering the clinic.

“I think multiple sclerosis is a good example of how good science and rigorously controlled clinical trials are bringing wonderful promises to patients,” remarked Dr. Fossati. “There are some MS patients that do not really respond to any of the available therapies, and for these people bone marrow transplantation can be really effective. So you take out their own bone marrow cells, and then you put them back. In this process, there is a reset of the immune cells that are inside the blood, the new blood cells that are formed no longer attack brain cells.”

“But, this took many decades and several different trials because the procedure also requires chemotherapy,” she continued. “When you take out the cells, you have to destroy everything else that is left inside, and this is what chemo does. It can have really strong side effects and it’s a very long procedure, which is why despite remarkable results, it’s not a first-line treatment. We have to evaluate whether it makes sense on a case-by-case basis to determine if the side effects are worth it.”

Dr. Fossati stresses that while it can be difficult, patience is key.

“As a patient I really understand how much we would like things to be working today, and we don’t want to wait ten years, but the science cannot be done in a day,” she said. “We have to collect a lot of data from a lot of people before we can say if a therapy works or not. What’s safe for one person might not be safe for everyone, so we have to be patient.”

What red flags can you look for when considering a stem cell therapy, and what questions can you ask?

“Many clinics are now using what I call ‘tokens of legitimacy,’” remarked Mr. Caulfield. “Their website looks spectacular. They’ll talk about their impressive board. They’ll talk about a clinical trial that they’re doing, and they might even register that clinical trial. They’ll refer to publications, maybe unrelated to what they’re doing, or published in what we call a ‘predatory journal’ where it’s a publication that is basically purchased.”

“So you can ask, what does the body of evidence say about how efficacious this is? Are they relying on testimonials almost entirely? Are they claiming that these stem cells can treat a whole bunch of different things? That’s not the way medicine works, right? If it can cure everything from your wrinkles to MS, then that’s a huge red red flag.”

“Another red flag is whether you’re paying for the treatment,” added Dr. Marks. “If you are being charged, it should be an approved product, and they should be able to explain to you why they are able to market it as such. You can always ask if they have a protocol with the FDA.”

The panelists stressed the importance of talking to someone in the field who you trust.

“Talk to someone you trust who has knowledge,” advised Dr. Kahn. “That to me is the most important thing: find someone in healthcare who you trust, and ask them.”

How can those in healthcare protect patients from being preyed upon by these clinics?

“The best defense here is information. Ask questions,” remarked Dr. Marks.

“If you really are concerned, you can send us (the FDA) an email or what can be even more effective is going to the state’s board of health or board of medical examiners,” he continued. “What I have found generally in my years as a regulator is that legitimate operators don’t mind when FDA comes sniffing around because they actually want to do the right thing and they’re very happy to work collaboratively with us. Whereas those doing unscrupulous practices don’t like to see us.”

How do we stay informed and protected?

“I think this is a complex problem, and we’re going to have to come at it from absolutely every direction: with the FDA, FTC, licensing boards, etc.,” remarked. Mr. Caulfield. “Also, we can get on social media and debunk this stuff. Debunking does work, so let’s get out there and highlight what the science actually says. 

“The International Society for Stem Cell Research (ISSCR) has a great website that people can go to that has really basic information about what’s available and what to look for,” he continued. “And if there was something out there and it was super efficacious, I promise, you’d know.”

“Ask the questions that you need,” agreed Dr. Marks. “And if somebody can’t answer them to your satisfaction, then ask elsewhere.”

“I just want to talk to the patients that are listening to us today,” remarked Dr. Fossati. “When you or your children are affected by disease, you completely lose your rationality. Don’t make a decision by yourself. You need to trust your doctor, and always work with a doctor that has followed you through the disease or with somebody else that you trust that knows the field. Ask for different opinions.”

“If something is available and working, multiple people should be able to tell you about an approved treatment. So be very cautious, and I know it’s hard. Only when you are in that situation can you understand, but that’s why you need to have people you trust.”

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