Why people seek out unproven and potentially unsafe stem cell treatments

Every day I field phone calls and emails from people looking for a stem cell therapy to help them cope with everything from arthritis to cancer. Often, they will mention that they saw an ad for a clinic online or in a local newspaper claiming they had stem cell therapies that could help fix anything and asking me if they are legitimate.

Even after I try to explain that the therapies these clinics are offering haven’t been tested in a clinical trial and that there’s scant evidence to show they are even safe let alone effective, I know that a good chunk of the callers are going to try them anyway.

Now a survey by the Mayo Clinic takes a deeper dive into why people are willing to put science aside and open up their wallets to go to predatory stem cell clinics for so-called “therapies”.

Dr. Zubin Master. Photo courtesy Mayo Clinic

In a news release Dr. Zubin Master, a co-author of the study, says many patients are lured in by hype and hope.

“We learned that many patients interested in stem cells had beliefs that are not supported by current medical evidence. For example, many thought stem cells were better than surgery or the standard of care.”

The survey asked 533 people, who had approached the Mayo Clinic’s Regenerative Medicine Therapeutic Suites for a consultation about arthritis or musculoskeletal problems, three questions.

  • Why are you interested in stem cell treatment for your condition?
  • How did you find out about stem cell treatment for your condition?
  • Have you contacted a stem cell clinic?

A whopping 46 percent of those who responded said they thought stem cell therapy would help them avoid or at least delay having to get a hip or knee replacement, or that it was a better option than surgery. Another 26 percent said they thought it would ease the pain of an arthritic joint.

The fact that there is little or no evidence to support any of these beliefs didn’t seem to matter. Most people say they got their information about these “therapies” online or by talking to friends and family.

These “therapies” aren’t cheap either. They can cost thousands, sometimes tens of thousands of dollars, and that comes out of the patient’s pocket because none of this is covered by insurance. Yet every year people turn to these bogus clinics because they don’t like the alternatives, mainly surgery.

There is a lot of promising stem cell research taking place around the US trying to find real scientific solutions to arthritic joints and other problems. The California Institute for Regenerative Medicine (CIRM) has invested almost $24 million in this research. But until those approaches have proven themselves effective and, hopefully, been approved for wider use by the Food and Drug Administration, CIRM and other agencies will have to keep repeating a message many people just don’t want to hear, that these therapies are not yet ready for prime time.

It’s hard to be modest when people keep telling you how good you are

THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST

I have a confession. Deep down I’m shallow. So when something I am part of is acknowledged as one of the best, I delight in it (my fellow bloggers Katie and Esteban also delight in it, I am just more shameless about letting everyone know.)

And that is just what happened with this blog, The Stem Cellar. We have been named as one of the “22 best biology and stem cell blogs of 2022”. And not just by anyone. We were honored by Dr. Paul Knoepfler, a stem cell scientist, avid blogger and all-round renaissance man (full disclosure, Paul is a recipient of CIRM funding but that has nothing to do with this award. Obviously.)

We are particularly honored to be on the list because Paul includes some heavy hitters including The Signals Blog, a site that he describes this way:

“This one from our friends in Canada is fantastic. They literally have dozens of authors, which is probably the most of any stem cell-related website, and their articles include many interesting angles. They post really often too. I might rank Signal and The Stem Cellar as tied for best stem cell blog in 2021.”

Now I’m really blushing.

Other highly regarded blogs are EuroStemCell, the Mayo Clinic Regenerative Medicine Blog and Stem Cell Battles (by Don Reed, a good friend of CIRM’s)

Another one of the 22 is David Jensen’s California Stem Cell report which is dedicated to covering the work of, you guessed it, CIRM. So, not only are we great bloggers, we are apparently great to blog about. 

As a further demonstration of my modesty I wanted to point out that Paul regularly produces ‘best of’ lists, including his recent “50 influencers on stem cells on Twitter to follow” which we were also on.

Call for a worldwide approach to regulating predatory stem cell clinics

You can’t fix a global problem at the local level. That’s the gist of a new perspective piece in the journal Stem Cell Reports that calls for a global approach to rogue stem cell clinics that offer bogus therapies.

The authors of the article are calling on the World Health Organization (WHO) to set up an advisory committee to draw up rules and regulations to help guide countries trying to shut these clinics down.

In a news release, senior author Mohamed Abou-el-Enein, the executive director of the joint University of Southern California/Children’s Hospital of Los Angeles Cell Therapy Program, says these clinics are trying to cash in on the promise of regenerative medicine.

“Starting in the early 2000s… unregulated stem cell clinics offering untested and poorly characterized treatments with insufficient information on their safety and efficacy began emerging all over the world, taking advantage of the media hype around stem cells and patients’ hope and desperation.”

Dr. Larry Goldstein

The authors include Lawrence Goldstein, PhD, a CIRM Board member and a Science Policy Fellows for the International Society for Stem Cell Research (ISSCR).

Zubin Master, an associate professor of biomedical ethics at the Mayo Clinic, says the clinics prey on vulnerable people who have serious medical conditions and who have often tried conventional medical approaches without success.

“We should aim to develop pathways to provide patients with evidenced-based experimental regenerative intervention as possible options where there is oversight, especially in circumstances where there is no suitable alternative left.”

The report says: “The unproven SCI (stem cell intervention) industry threatens the advancement of regenerative medicine. Reports of adverse events from unproven SCIs has the potential to affect funding and clinical trial recruitment, as well as increasing burdens among regulatory agencies to oversee the industry.

Permitting unregulated SCIs to flourish demonstrates a lack of concern over patient welfare and undermines the need for scientific evidence for medicinal product R&D. While some regulatory agencies have limited oversight or enforcement powers, or choose not to use them, unproven SCI clinics still serve to undermine authority given to regulatory agencies and may reduce public trust impacting the development of safe and effective therapies. Addressing the continued proliferation of clinics offering unproven SCIs is a problem worth addressing now.”

The authors say the WHO is uniquely positioned to help create a framework for the field that can help address these issues. They recommend setting up an advisory committee to develop global standards for regulations governing these clinics that could be applied in all countries. They also say we need more educational materials to let physicians as well as patients understand the health risks posed by bogus clinics.

This article comes out in the same week that reports by the Pew Charitable Trust and the FDA also called for greater regulation of these predatory clinics (we blogged about that here). Clearly there is growing recognition both in the US and worldwide that these clinics pose a threat not just to the health and safety of patients, but also to the reputation of the field of regenerative medicine as a whole.

“I believe that the global spread of unproven stem cell therapies reflects critical gaps in the international system for responding to health crises, which could put the life of thousands of patients in danger,” Abou-el-Enein says. “Urgent measures are needed to enhance the global regulatory capacity to detect and respond to this eminent crisis rapidly.”

Of Mice and Men, and Women Too; Stem cell stories you might have missed

Mice brains can teach us a lot

Last week’s news headlines were dominated by one big story, the use of a stem cell transplant to effectively cure a person of HIV. But there were other stories that, while not quite as striking, did also highlight how the field is advancing.

A new way to boost brain cells (in mice!)

It’s hard to fix something if you don’t really know what’s wrong in the first place. It would be like trying to determine why a car is not working just by looking at the hood and not looking inside at the engine. The human brain is far more complex than a car so trying to determine what’s going wrong is infinitely more challenging. But a new study could help give us a new option.

Researchers in Luxembourg and Germany have developed a new computer model for what’s happening inside the brain, identifying what cells are not operating properly, and fixing them.

Antonio del Sol, one of the lead authors of the study – published in the journal Cell – says their new model allows them to identify which stem cells are active and ready to divide, or dormant. 

“Our results constitute an important step towards the implementation of stem cell-based therapies, for instance for neurodegenerative diseases. We were able to show that, with computational models, it is possible to identify the essential features that are characteristic of a specific state of stem cells.”

The work, done in mice, identified a protein that helped keep brain stem cells inactive in older animals. By blocking this protein they were able to help “wake up” those stem cells so they could divide and proliferate and help regenerate the aging brain.

And if it works in mice it must work in people right? Well, that’s what they hope to see next.

Deeper understanding of fetal development

According to the Mayo Clinic between 10 and 20 percent of known pregnancies end in miscarriage (though they admit the real number may be even higher) and our lack of understanding of fetal development makes it hard to understand why. A new study reveals a previously unknown step in this development that could help provide some answers and, hopefully, lead to ways to prevent miscarriages.

Researchers at the Karolinska Institute in Sweden used genetic sequencing to follow the development stages of mice embryos. By sorting those different sequences into a kind of blueprint for what’s happening at every stage of development they were able to identify a previously unknown phase. It’s the time between when the embryo attaches to the uterus and when it begins to turn these embryonic stem cells into identifiable parts of the body.

Qiaolin Deng, Karolinska Institute

Lead researcher Qiaolin Deng says this finding provides vital new evidence.

“Being able to follow the differentiation process of every cell is the Holy Grail of developmental biology. Knowledge of the events and factors that govern the development of the early embryo is indispensable for understanding miscarriages and congenital disease. Around three in every 100 babies are born with fetal malformation caused by faulty cellular differentiation.”

The study is published in the journal Cell Reports.

Could a new drug discovery reduce damage from a heart attack?

Every 40 seconds someone in the US has a heart attack. For many it is fatal but even for those who survive it can lead to long-term damage to the heart that ultimately leads to heart failure. Now British researchers think they may have found a way to reduce that likelihood.

Using stem cells to create human heart muscle tissue in the lab, they identified a protein that is activated after a heart attack or when exposed to stress chemicals. They then identified a drug that can block that protein and, when tested in mice that had experienced a heart attack, they found it could reduce damage to the heart muscle by around 60 percent.

Prof Michael Schneider, the lead researcher on the study, published in Cell Stem Cell, said this could be a game changer.

“There are no existing therapies that directly address the problem of muscle cell death and this would be a revolution in the treatment of heart attacks. One reason why many heart drugs have failed in clinical trials may be that they have not been tested in human cells before the clinic. Using both human cells and animals allows us to be more confident about the molecules we take forward.”

Stem cells and professional sports: a call for more science and less speculation

In the world of professional sports, teams invest tens of millions of dollars in players. Those players are under intense pressure to show a return on that investment for the team, and that means playing as hard as possible for as long as possible. So it’s no surprise that players facing serious injuries will often turn to any treatment that might get them back in the game.

image courtesy Scientific American

image courtesy Scientific American

A new study published last week in 2014 World Stem Cell Report (we blogged about it here) highlighted how far some players will go to keep playing, saying at least 12 NFL players have undergone unproven stem cell treatments in the last five years. A session at the recent World Stem Cell Summit in San Antonio, Texas showed that football is not unique, that this is a trend in all professional sports.

Dr. Shane Shapiro, an orthopedic surgeon at the Mayo Clinic, says it was an article in the New York Times in 2009 about two of the NFL players named in the World Stem Cell Report that led him to becoming interested in stem cells. The article focused on two members of the Pittsburgh Steelers team who were able to overcome injuries and play in the Super Bowl after undergoing stem cell treatment, although there was no direct evidence the stem cells caused the improvement.

“The next day, the day after the article appeared, I had multiple patients in my office with copies of the New York Times asking if I could perform the same procedure on them.”

Dr. Shapiro had experienced what has since become one of the driving factors behind many people seeking stem cell therapies, even ones that are unproven; the media reports high profile athletes getting a treatment that seems to work leading many non-athletes to want the same.

“This is not just about high profile athletes it’s also about older patients, weekend warriors and all those with degenerative joint disease, which affects around 50 million Americans. Currently for a lot of these degenerative conditions we don’t have many good non- surgical options, basically physical therapy, gentle pain relievers or steroid injections. That’s it. We have to get somewhere where we have options to slow down this trend, to slow down the progression of these injuries and problems.”

Shapiro says one of the most popular stem cell-based approaches in sports medicine today is the use of plasma rich platelets or PRP. The idea behind it makes sense, at least in theory. Blood contains platelets that contain growth factors that have been shown to help tissue heal. So injecting a patient’s platelets into the injury site might speed recovery and, because it’s the patient’s own platelets, the treatment probably won’t cause any immune response or prove to be harmful.

That’s the theory. The problem is few well-designed clinical trials have been done to see if that’s actually the case. Shapiro talked about one relatively small, non-randomized study that used PRP and in a 14-month follow-up found that 83% of patients reported feeling satisfied with their pain relief. However, 84% of this group did not have any visible improved appearance on ultrasound.

He is now in the process of carrying out a clinical trial, approved by the Food and Drug Administration (FDA), using bone marrow aspirate concentrate (BMAC) cells harvested from the patient’s own bone marrow. Because those cells secrete growth factors such as cytokines and chemokines they hope they may have anti-inflammatory and regenerative properties. The cells will be injected into 25 patients, all of whom have arthritic knees. They hope to have results next year.

Dr. Paul Saenz is a sports medicine specialist and the team physician for the San Antonio Spurs, the current National Basketball Association champions. He says that sports teams are frequently criticized for allowing players to undergo unproven stem cell treatments but he says it’s unrealistic to expect teams to do clinical studies to see if these therapies work, that’s not their area of expertise. But he also says team physicians are very careful in what they are willing to try.

“As fervent as we are to help bring an athlete back to form, we are equally fervent in our desire not to harm a $10 million athlete. Sports physicians are very conservative and for them stem cells are never the first thing they try, they are options when other approaches have failed.”

Saenz said while there are not enough double blind, randomized controlled clinical trials he has seen many individual cases, anecdotal evidence, where the use of stem cells has made a big difference. He talked about one basketball player, a 13-year NBA veteran, who was experiencing pain and mobility problems with his knee. He put the player on a biologic regimen and performed a PRP procedure on the knee.

“What we saw over the next few years was decreased pain, and a dramatic decrease in his reliance on non-steroidal anti inflammatory drugs. We saw improved MRI findings, improved athletic performance with more time on court, more baskets and more rebounds.”

But Saenz acknowledges that for the field to advance anecdotal stories like this are not enough, well-designed clinical trials are needed. He says right now there is too much guesswork in treatments, that there is not even any agreement on best practices or standardized treatment protocols.

Dr. Shapiro says for too long the use of stem cells in sports medicine has been the realm of individual physicians or medical groups. That has to change:

“If we are ever to move forward on this it has to be opened up to the scientific community, we have to do the work, do the studies, complete the analysis, open it up to our peers, report it in a reputable journal. If we want to treat the 50 million Americans who need this kind of therapy we need to go through the FDA approval process. We can’t just continue to treat the one patient a month who can afford to pay for all this themselves. “