stem cell clinics

California Stem Cell and Regenerative Therapy Task Force holds meeting to consider options for patient protection

/ Yimy Villa / 1 Comment
Dr. Maria Millan, President and CEO of CIRM, attended the meeting to discuss the importance of having systems in place for patient protection.

What procedures are in place to ensure the quality and safety of stem cell treatments? How can patients guard against deceptive promotional practices for treatments that have no basis in science? What new procedures are needed to support patients and the development of new treatments?

These questions and others were discussed this past Wednesday by the Medical Board of California’s Stem Cell and Regenerative Therapy Task Force. The task force  held an interested parties meeting to receive information and input on options to promote consumer protection.

CIRM, the Alpha Stem Cell Clinic Network, and the Department of Public Health gave formal presentations to the task force.

Dr. Maria Millan started by providing the task force with an overview of the field in general and the 56 CIRM funded Clinical Trials to illustrate the enormous promise of the field. She then contrasted this promise against numerous reports of patients being harmed by unproven and unregulated stem cell treatments provided by practitioners operating outside their field of training. Dr. Millan emphasized the critical importance of having systems in place to provide assurance to patients that treatments are appropriate for their particular disease.  She elaborated on CIRM’s core mantra that stem cell treatments be regulated, reputable and reliable. We discussed the three Rs in this posting. The fundamental aim is to put the patient interests at the center of a system that meets all regulatory and professional standards of care.

Dr. Mehrdad Abedi, Director of the UC Davis Alpha Stem Cell Clinic provided concrete examples of how they are implementing the 3Rs in their operations. Dr. Abedi emphasized the importance of best practices for manufacturing and processing stem cell products and for clinical care. He cited the operations at the UC Davis Institute for Regenerative Cures and the various oversight committees tasked with protecting the rights and interests of patients.  Collectively, this approach, embraced by all the CIRM Alpha Stem Cell Clinics, serves to ensure all clinical trials regulated, reputable and reliable.

State of the art materials processing at the UC Davis Center for Regenerative Cures

Dr. Charity Dean of the Department of Public Health described the role of the Food and Drug Branch in licensing facilities involved in the preparation, processing and labeling of drugs. This authority extends to facilities outside of California that ship products into the state. Dr. Dean illustrated how the Department of Public Health’s Food and Drug Branch licenses manufactures, and this licensing system is designed to protect patients using such products.

After discussion and public comment, the task force co-chair, Dr. Krauss suggested the Medical Board would consider options for patient protection, include:

  • Guidance and education materials for medical practitioners
  • Sample informed consent documents designed to inform patients about the potential risks and benefits of stem cell treatments
  • Public education materials
  • An adverse event reporting system

Taking the message to the people: fighting for the future of stem cell research in California

/ Kevin McCormack / Leave a comment

Stem cells have been in the news a lot this week, and not necessarily for the right reason.

First, the US Food and Drug Administration (FDA) won a big legal decision in its fight to crack down on clinics offering bogus, unproven and unapproved stem cell therapies.

William Shatner: Photograph by Jerry Avenaim.wikimedia.org/w/index.php?curid=3413082

But then came news that another big name celebrity, in this case Star Trek star William Shatner, was going to one of these clinics for an infusion of what he called “restorative cells”.

It’s a reminder that for every step forward we take in trying to educate the public about the dangers of clinics offering unproven therapies, we often take another step back when a celebrity essentially endorses the idea.

So that’s why we are taking our message directly to the people, as often as we can and wherever we can.

In June we are going to be holding a free, public event in Los Angeles to coincide with the opening of the International Society for Stem Cell Research’s Annual Conference, the biggest event on the global stem cell calendar. There’s still time to register for that by the way. The event is from 6-7pm on Tuesday, June 25th in Petree Hall C., at the Los Angeles Convention Center at 1201 South Figueroa Street, LA 90015.

The event is open to everyone and it’s FREE. We have created an Eventbrite page where you can get all the details and RSVP if you are coming.

It’s going to be an opportunity to learn about the real progress being made in stem cell research, thanks in no small part to CIRM’s funding. We’re honored to be joined by UCLA’s Dr. Don Kohn, who has helped cure dozens of children born with a fatal immune system disorder called severe combined immunodeficiency, also known as “bubble baby disease”. And we’ll hear from the family of one of those children whose life he helped save.

And because CIRM is due to run out of money to fund new projects by the end of this year you’ll also learn about the very real concerns we have about the future of stem cell research in California and what can be done to address those concerns. It promises to be a fascinating evening.

But that’s not all. Our partners at USC will be holding another public event on stem cell research, on Wednesday June 26th from 6.30p to 8pm. This one is focused on treatments for age-related blindness. This features some of the top stem cell scientists in the field who are making encouraging progress in not just slowing down vision loss, but in some cases even reversing it.

You can find out more about that event here.

We know that we face some serious challenges in trying to educate people about the risks of going to a clinic offering unproven therapies. But we also know we have a great story to tell, one that shows how we are already changing lives and saving lives, and that with the support of the people of California we’ll do even more in the years to come.

Media matters in spreading the word

/ Kevin McCormack / 2 Comments
Cover of New Yorker article on “The Birth Tissue Profiteers”. Illustration by Ben Jones

When you have a great story to tell the best and most effective way to get it out to the widest audience is still the media, both traditional mainstream and new social media. Recently we have seen three great examples of how that can be done and, hopefully, the benefits that can come from it.

First, let’s go old school. Earlier this month Caroline Chen wrote a wonderful in-depth article about clinics that are cashing in on a gray area in stem cell research. The piece, a collaboration between the New Yorker magazine and ProPublica, focused on the use of amniotic stem cell treatments and the gap between what the clinics who offer it are claiming it can do, and the reality.

Here’s one paragraph profiling a Dr. David Greene, who runs a company providing amniotic fluid to clinics. It’s a fine piece of writing showing how the people behind these therapies blur the lines between fact and reality, not just about the cells but also about themselves:

“Greene said that amniotic stem cells derive their healing power from an ability to develop into any kind of tissue, but he failed to mention that mainstream science does not support his claims. He also did not disclose that he lost his license to practice medicine in 2009, after surgeries he botched resulted in several deaths. Instead, he offered glowing statistics: amniotic stem cells could help the heart beat better, “on average by twenty per cent,” he said. “Over eighty-five per cent of patients benefit exceptionally from the treatment.”

Greene later backpedals on that claim, saying:

“I don’t claim that this is a treatment. I don’t claim that it cures anything. I don’t claim that it’s a permanent fix. All I discuss is maybe, potentially, people can get some improvements from stem-cell care.”

CBS2 TV Chicago

This week CBS2 TV in Chicago did their own investigative story about how the number of local clinics offering unproven and unapproved therapies is on the rise. Reporter Pam Zekman showed how misleading newspaper ads brought in people desperate for something, anything, to ease their arthritis pain.

She interviewed two patients who went to one of those clinics, and ended up out of pocket, and out of luck.

“They said they would regenerate the cartilage,” Patricia Korona recalled. She paid $4500 for injections in her knee, but the pain continued. Later X-rays were ordered by her orthopedic surgeon.

“He found bone on bone,” Korona said. “No cartilage grew, which tells me it failed; didn’t work.”

John Zapfel paid $14,000 for stem cell injections on each side of his neck and his shoulder. But an MRI taken by his current doctor showed no improvement.

“They ripped me off, and I was mad.” Zapfel said.      

TV and print reports like this are a great way to highlight the bogus claims made by many of these clinics, and to shine a light on how they use hype to sell hope to people who are in pain and looking for help.

At a time when journalism seems to be increasingly under attack with accusations of “fake news” it’s encouraging to see reporters like these taking the time and news outlets devoting the resources to uncover shady practices and protect vulnerable patients.

But the news isn’t all bad, and the use of social media can help highlight the good news.

That’s what happened yesterday in our latest CIRM Facebook Live “Ask the Stem Cell Team” event. The event focused on the future of stem cell research but also included a really thoughtful look at the progress that’s been made over the last 10-15 years.

We had two great guests, UC Davis stem cell researcher and one of the leading bloggers on the field, Paul Knoepfler PhD; and David Higgins, PhD, a scientist, member of the CIRM Board and a Patient Advocate for Huntington’s Disease. They were able to highlight the challenges of the early years of stem cell research, both globally and here at CIRM, and show how the field has evolved at a remarkable rate in recent years.

Paul Knoepfler

Naturally the subject of the “bogus clinics” came up – Paul has become a national expert on these clinics and is quoted in the New Yorker article – as did the subject of the frustration some people feel at what they consider to be the too-slow pace of progress. As David Higgins noted, we all think it’s too slow, but we are not going to race recklessly ahead in search of something that might heal if we might also end up doing something that might kill.

David Higgins

A portion of the discussion focused on funding and, in particular, what happens if CIRM is no longer around to fund the most promising research in California. We are due to run out of funding for new projects by the end of this year, and without a re-infusion of funds we will be pretty much closing our doors by the end of 2020. Both Paul and David felt that could be disastrous for the field here in California, depriving the most promising projects of support at a time when they needed it most.

It’s probably not too surprising that three people so closely connected to CIRM (Paul has received funding from us in the past) would conclude that CIRM is needed for stem cell research to not just survive but thrive in California.

A word of caution before you watch: fashion conscious people may be appalled at how my pocket handkerchief took on a life of its own.

Media shine a spotlight on dodgy stem cell clinics

/ Kevin McCormack / Leave a comment
A doctor collects fat from a patient’’s back as part of an experimental stem cell procedure in Beverly Hills, Calif. on Dec. 5, 2014. (Raquel Maria Dillon / Associated Press)

For several years now, we have been trying to raise awareness about the risks posed by clinics offering unproven or unapproved stem cell therapies. At times it felt as if we were yelling into the wind, that few people were listening. But that’s slowly changing. A growing number of TV stations and newspapers are picking up the message and warning their readers and viewers. It’s a warning that is getting national exposure.

Why are we concerned about these clinics? Well, they claim their therapies, which usually involve the patient’s own fat or blood cells, can cure everything from arthritis to Alzheimer’s. However, they offer no scientific proof, have no studies to back up their claims and charge patients thousands, sometimes tens of thousands of dollars.

In the LA Times, for example, reporter Usha Lee McFarling, wrote an article headline “California has gone crazy for sketchy stem cell treatments”. In it she writes about the claims made by these clinics and the dangers they pose:

“If it sounds too good to be true, it is. There is no good scientific evidence the pricey treatments work, and there is growing evidence that some are dangerous, causing blindness, tumors and paralysis. Medical associations, the federal government and even Consumer Reports have all issued stern warnings to patients about the clinics.”

In Denver, the ABC TV station recently did an in-depth interview with a local doctor who is trying to get Colorado state legislators to take legal action against stem cell clinics making these kinds of unsupported claims.

Chris Centeno of the Centeno-Schultz Clinic, who’s specialized in regenerative medicine and research for more than a decade, said too many people are simply being scammed.

“It’s really out of control,” he told the station.

ABC7 did a series of reports last year on the problem and that may be prompting this push for a law warning consumers about the dangers posed by these unregulated treatments which are advertised heavily online, on TV and in print.

In California there is already one law on the books attempting to warn consumers about these clinics. CIRM worked with State Senator Ed Hernandez to get that passed (you can read about that here) and we are continuing to support even stronger measures.

And the NBC TV station in San Diego recently reported on the rise of stem cell clinics around the US, a story that was picked up by the networks and run on the NBC Today Show.

One of the critical elements in helping raise awareness about the issue has been the work done by Paul Knoepfler and Leigh Turner in identifying how many of these clinics there are around the US. Their report, published in the journal Cell Stem Cell, was the first to show how big the problem is. It attracted national attention and triggered many of the reports that followed.

It is clear momentum is building and we hope to build on that even further. Obviously, the best solution would be to have the Food and Drug Administration (FDA) crack down on these clinics, and in some cases they have. But the FDA lacks the manpower to tackle all of them.

That’s where the role of the media is so important. By doing stories like these and raising awareness about the risks these clinics pose they can hopefully help many patients avoid treatments that will do little except make a dent in their pocket.

Headline: Stem Cell Roundup: Here are some stem cell stories that caught our eye this past week.

/ Kevin McCormack / 1 Comment

In search of a miracle

Jordan and mother

Luane Beck holds Jordan in the emergency room while he suffers a prolonged seizure. Jordan’s seizures sometimes occur one after another with no break, and they can be deadly without emergency care. Photo courtesy San Francisco Chronicle’s Kim Clark

One of the toughest parts of my job is getting daily calls and emails from people desperate for a stem cell treatment or cure for themselves or a loved one and having to tell them that I don’t know of any. You can hear in their voice, read it in their emails, how hard it is for them to see someone they love in pain or distress and not be able to help them.

I know that many of those people may think about turning to one of the many stem cell clinics, here in the US and in Mexico and other countries, that are offering unproven and unapproved therapies. These clinics are offering desperate people a sense of hope, even if there is no evidence that the therapies they provide are either safe or effective.

And these “therapies” come with a big cost, both emotional and financial.

The San Francisco Chronicle this week launched the first in a series of stories they are doing about stem cells and stem cell research, the progress being made and the problems the field still faces.

One of the biggest problems, are clinics that offer hope, at a steep price, but no evidence to show that hope is justified. The first piece in the Chronicle series is a powerful, heart breaking story of one mother’s love for her son and her determination to do all she can to help him, and the difficult, almost impossible choices she has to make along the way.

It’s called: In search of a miracle.

A little turbulence, and a French press-like device, can help boost blood platelet production

Every year more than 21 million units of blood are transfused into people in the US. It’s a simple, life-saving procedure. One of the most important elements in transfusions are  platelets, the cells that stop bleeding and have other healing properties. Platelets, however, have a very short shelf life and so there is a constant need to get more from donors. Now a new study from Japan may help fix that problem.

Platelets are small cells that break off much larger cells called megakaryocytes. Scientists at the Center for iPS Cell Research and Application (CiRA) created billions of megakaryocytes using iPS technology (which turns ordinary cells into any other kind of cell in the body) and then placed them in a bioreactor. The bioreactor then pushed the cells up and down – much like you push down on a French press coffee maker – which helped promote the generation of platelets.

In their study, published in the journal Cell, they report they were able to generate 100 billion platelets, enough to be able to treat patients.

In a news release, CiRA Professor Koji Eto said they have shown this works in mice and now they want to see if it also works in people:

“Our goal is to produce platelets in the lab to replace human donors.”

Stem Cell Photo of the Week 

Photo Jul 11, 6 00 19 PM

Students at the CIRM Bridges program practice their “elevator pitch”. Photo Kyle Chesser

This week we held our annual CIRM Bridges to Stem Cell Research conference in Newport Beach. The Bridges program provides paid internships for undergraduate and masters-level students, a chance to work in a world-class stem cell research facility and get the experience needed to pursue a career in science. The program is training the next generation of stem cell scientists to fill jobs in California’s growing stem cell research sector.

This year we got the students to practice an “elevator Pitch”, a 30 second explanation, in plain English, of what they do, why they do it and why people should care. It’s a fun exercise but also an important one. We want scientists to be able to explain to the public what they are doing and why it’s important. After all, the people of California are supporting this work so they have a right to know, in language they can understand, how their money is changing the face of medicine.

Stem cell clinics make big claims but offer little evidence they can treat osteoarthritic knees

/ Kevin McCormack / 3 Comments

osteoarthritis knee

If someone says they have a success rate of close to 100 percent in treating a major health problem but offer little evidence to back that up, you might be excused for being more than a tad skeptical. And a new study says you would be right.

The health problem in question is osteoarthritis (OA) of the knee, something that affects almost 10 million Americans. It’s caused by the wearing down of the protective cartilage in the knee. That cartilage acts as a kind of shock absorber, so when it’s gone you have bone rubbing against bone. That’s not just painful but also debilitating, making it hard to lead an active life.

There is a lot of research taking place – including a clinical trial that CIRM is funding – that focuses on using stem cells to create new cartilage, but so far nothing has been approved by the US Food and Drug Administration for wider use. The reason for that is simple. No approach has yet proven it is both safe and effective.

No evidence? No worries

But that doesn’t stop many clinics around the US, and around the world, from claiming they have treatments that work and charging patients a hefty sum to get them.

In a study presented at the Annual Meeting of the American Academy of Orthopaedic Surgeons, researchers contacted 317 clinics in the US that directly market stem cell therapies to consumers. They asked the clinics for information on the cost of the procedure and their success rate.

  • Only 65 clinics responded
  • Lowest price was $1,150
  • Highest price was $12,000,
  • Average price of $5,156.

Only 36 clinics responded with information about success rates.

  • 10 claimed between 90 and 100 percent success
  • 15 claimed 80 to 90 percent success
  • 10 claimed 70 to 80 percent
  • One said just 55 percent.

None offered any evidence based on a clinical trial that supported those claims, and there was no connection between how much they charged and how successful they claimed to be.

In a news release about the study – which appears in the Journal of Knee Surgery – George Muschler, one of the lead authors, said that orthopedic surgeons have a duty to give patients the best information available about all treatment options.

“Recent systematic reviews of cellular therapies for the treatment of knee OA (over 400 papers screened) have found poor levels of evidence for the efficacy of these treatments to date. Current evidence does not justify the rapid rate of growth for these therapies.”

Nicolas Piuzzi, the other lead author on the study, says if the evidence doesn’t justify the growth in the number of clinics offering these therapies, it certainly doesn’t justify the prices they charge.

“The claim of “stem cell” therapy carries a high level of expectations for the potential benefits, but research is still many years away from providing clear evidence of effective treatment to patients. As clinicians and researchers, we have ethical, scientific, legal and regulatory concerns. Patients need to be aware of the status of research within the field. If they receive information from anyone offering a treatment claim of an 80 to 100 percent successful recovery, they should be concerned in observance of published peer-reviewed evidence.”

Stem Cell Stories that Caught Our Eye: New law to protect consumers; using skin to monitor blood sugar; and a win for the good guys

/ Kevin McCormack / Leave a comment

Hernendez

State Senator Ed Hernandez

New law targets stem cell clinics that offer therapies not approved by the FDA

For some time now CIRM and others around California have been warning consumers about the risks involved in going to clinics that offer stem cell therapies that have not been tested in a clinical trial or approved by the U.S. Food and Drug Administration (FDA) for use in patients.

Now a new California law, authored by State Senator Ed Hernandez (D-West Covina) attempts to address that issue. It will require medical clinics whose stem cell treatments are not FDA approved, to post notices and provide handouts to patients warning them about the potential risk.

In a news release Sen. Hernandez said he hopes the new law, SB 512, will protect consumers from early-stage, unproven experimental therapies:

“There are currently over 100 medical offices in California providing non-FDA approved stem cell treatments. Patients spend thousands of dollars on these treatments, but are totally unaware of potential risks and dangerous side effects.”

Sen. Hernandez’s staffer Bao-Ngoc Nguyen crafted the bill, with help from CIRM Board Vice Chair Sen. Art Torres, Geoff Lomax and UC Davis researcher Paul Knoepfler, to ensure it targeted only clinics offering non-FDA approved therapies and not those offering FDA-sanctioned clinical trials.

For example the bill would not affect CIRM’s Alpha Stem Cell Clinic Network because all the therapies offered there have been given the green light by the FDA to work with patients.

Blood_Glucose_Testing 

Using your own skin as a blood glucose monitor

One of the many things that people with diabetes hate is the constant need to monitor their blood sugar level. Usually that involves a finger prick to get a drop of blood. It’s simple but not much fun. Attempts to develop non-invasive monitors have been tried but with limited success.

Now researchers at the University of Chicago have come up with another alternative, using the person’s own skin to measure their blood glucose level.

Xiaoyang Wu and his team accomplished this feat in mice by first creating new skin from stem cells. Then, using the gene-editing tool CRISPR, they added in a protein that sticks to sugar molecules and another protein that acts as a fluorescent marker. The hope was that the when the protein sticks to sugar in the blood it would change shape and emit fluorescence which could indicate if blood glucose levels were too high, too low, or just right.

The team then grafted the skin cells back onto the mouse. When those mice were left hungry for a while then given a big dose of sugar, the skin “sensors” reacted within 30 seconds.

The researchers say they are now exploring ways that their findings, published on the website bioRxiv, could be duplicated in people.

While they are doing that, we are supporting ViaCytes attempt to develop a device that doesn’t just monitor blood sugar levels but also delivers insulin when needed. You can read about our recent award to ViaCyte here.

Deepak

Dr. Deepak Srivastava

Stem Cell Champion, CIRM grantee, and all-round-nice guy named President of Gladstone Institutes

I don’t think it would shock anyone to know that there are a few prima donnas in the world of stem cell research. Happily, Dr. Deepak Srivastava is not one of them, which makes it such a delight to hear that he has been appointed as the next President of the Gladstone Institutes in San Francisco.

Deepak is a gifted scientist – which is why we have funded his work – a terrific communicator and a really lovely fella; straight forward and down to earth.

In a news release announcing his appointment – his term starts January 1 next year – Deepak said he is honored to succeed the current President, Sandy Williams:

“I joined Gladstone in 2005 because of its unique ability to leverage diverse basic science approaches through teams of scientists focused on achieving scientific breakthroughs for mankind’s most devastating diseases. I look forward to continue shaping this innovative approach to overcome human disease.”

We wish him great success in his new role.

 

 

 

CIRM weekly stem cell roundup: stomach bacteria & cancer; vitamin C may block leukemia; stem cells bring down a 6’2″ 246lb football player

/ Kevin McCormack / Leave a comment

gastric

This is what your stomach glands looks like from the inside:  Credit: MPI for Infection Biology”

Stomach bacteria crank up stem cell renewal, may be link to gastric cancer (Todd Dubnicoff)

The Centers for Disease Control and Prevention estimate that two-thirds of the world’s population is infected with H. pylori, a type of bacteria that thrives in the harsh acidic conditions of the stomach. Data accumulated over the past few decades shows strong evidence that H. pylori infection increases the risk of stomach cancers. The underlying mechanisms of this link have remained unclear. But research published this week in Nature suggests that the bacteria cause stem cells located in the stomach lining to divide more frequently leading to an increased potential for cancerous growth.

Tumors need to make an initial foothold in a tissue in order to grow and spread. But the cells of our stomach lining are replaced every four days. So, how would H. pylori bacterial infection have time to induce a cancer? The research team – a collaboration between scientists at the Max Planck Institute in Berlin and Stanford University – asked that question and found that the bacteria are also able to penetrate down into the stomach glands and infect stem cells whose job it is to continually replenish the stomach lining.

Further analysis in mice revealed that two groups of stem cells exist in the stomach glands – one slowly dividing and one rapidly dividing population. Both stem cell populations respond similarly to an important signaling protein, called Wnt, that sustains stem cell renewal. But the team also discovered a second key stem cell signaling protein called R-spondin that is released by connective tissue underneath the stomach glands. H. pylori infection of these cells causes an increase in R-spondin which shuts down the slowly dividing stem cell population but cranks up the cell division of the rapidly dividing stem cells. First author, Dr. Michal Sigal, summed up in a press release how these results may point to stem cells as the link between bacterial infection and increased risk of stomach cancer:

“Since H. pylori causes life-long infections, the constant increase in stem cell divisions may be enough to explain the increased risk of carcinogenesis observed.”

vitamin-c-1200x630

Vitamin C may have anti-blood cancer properties

Vitamin C is known to have a number of health benefits, from preventing scurvy to limiting the buildup of fatty plaque in your arteries. Now a new study says we might soon be able to add another benefit: it may be able to block the progression of leukemia and other blood cancers.

Researchers at the NYU School of Medicine focused their work on an enzyme called TET2. This is found in hematopoietic stem cells (HSCs), the kind of stem cell typically found in bone marrow. The absence of TET2 is known to keep these HSCs in a pre-leukemic state; in effect priming the body to develop leukemia. The researchers showed that high doses of vitamin C can prevent, or even reverse that, by increasing the activity level of TET2.

In the study, in the journal Cell, they showed how they developed mice that could have their levels of TET2 increased or decreased. They then transplanted bone marrow with low levels of TET2 from those mice into healthy, normal mice. The healthy mice started to develop leukemia-like symptoms. However, when the researchers used high doses of vitamin C to restore the activity levels of TET2, they were able to halt the progression of the leukemia.

Now this doesn’t mean you should run out and get as much vitamin C as you can to help protect you against leukemia. In an article in The Scientist, Benjamin Neel, senior author of the study, says while vitamin C does have health benefits,  consuming large doses won’t do you much good:

“They’re unlikely to be a general anti-cancer therapy, and they really should be understood based on the molecular understanding of the many actions vitamin C has in cells.”

However, Neel says these findings do give scientists a new tool to help them target cells before they become leukemic.

Jordan reed

Bad toe forces Jordan Reed to take a knee: Photo courtesy FanRag Sports

Toeing the line: how unapproved stem cell treatment made matters worse for an NFL player  

American football players are tough. They have to be to withstand pounding tackles by 300lb men wearing pads and a helmet. But it wasn’t a crunching hit that took Washington Redskins player Jordan Reed out of the game; all it took to put the 6’2” 246 lb player on the PUP (Physically Unable to Perform) list was a little stem cell injection.

Reed has had a lingering injury problem with the big toe on his left foot. So, during the off-season, he thought he would take care of the issue, and got a stem cell injection in the toe. It didn’t quite work the way he hoped.

In an interview with the Richmond Times Dispatch he said:

“That kind of flared it up a bit on me. Now I’m just letting it calm down before I get out there. I’ve just gotta take my time, let it heal and strengthen up, then get back out there.”

It’s not clear what kind of stem cells Reed got, if they were his own or from a donor. What is clear is that he is just the latest in a long line of athletes who have turned to stem cells to help repair or speed up recovery from an injury. These are treatments that have not been approved by the Food and Drug Administration (FDA) and that have not been tested in a clinical trial to make sure they are both safe and effective.

In Reed’s case the problem seems to be a relatively minor one; his toe is expected to heal and he should be back in action before too long.

Stem cell researcher and avid blogger Dr. Paul Knoepfler wrote he is lucky, others who take a similar approach may not be:

“Fortunately, it sounds like Reed will be fine, but some people have much worse reactions to unproven stem cells than a sore toe, including blindness and tumors. Be careful out there!”

‘Pay-to-Participate’ stem cell clinical studies, the ugly stepchild of ClinicalTrials.gov

/ Karen Ring / 10 Comments

When patients are looking for clinical trials testing new drugs or treatments for their disease, one of the main websites they visit is ClinicalTrials.gov. It’s a registry provided by the National Institutes of Health (NIH) of approximately 250,000 clinical trials spanning over 200 countries around the world.

ClinicalTrials.gov website

If you visit the website, you’ll find CIRM’s 28 active clinical trials testing stem cell-based therapies for indications like spinal cord injury, type 1 diabetes, heart failure, ALS, cancer and more. These are Food and Drug Administration (FDA)-approved trials, meaning that researchers did the proper preclinical studies to prove that a therapy was safe and effective in animal models and received approval from the US FDA to test the treatment in human clinical trials.

As the largest clinical registry in the world, ClinicalTrials.gov is a very valuable resource for patients and the public. But there are studies on the website that have recently surfaced and taken on the role of ‘ugly stepchild’. These are unapproved stem cell therapies from companies and stem cell clinics that are registering their “pay-to-participate treatments”. And they are doing so in clever ways that don’t make it obvious to patients that the trials aren’t legitimate. The reason this is so troubling is that unproven therapies can be dangerous or even life-threatening to patients.

Leigh Turner

Leigh Turner, an associate professor of bioethics at the University of Minnesota, has written extensively about the serious problem of stem cell clinics marketing unproven stem cell therapies to desperate patients. Turner, in collaboration with UC Davis professor Dr. Paul Knoepfler, published a study in Cell Stem Cell last year that identified over 550 clinics in the US that promote unproven treatments for almost any condition, including diseases like Alzheimer’s where research has shown that cures are a long way off.

Today, Turner published an article in Regenerative Medicine that shines a light on how companies and clinics are taking advantage of ClinicalTrials.gov to promote their “pay-to-participate” unproven stem cell studies. The article is available for free if you register with RegMedNet, but you can find news coverage about Turner’s piece through EurekAlert,  Wired Magazine and the San Diego Union Tribune.

In an interview with RegMedNet, Turner explained that his research into how businesses promote unproven stem cell therapies led to the discovery that these studies were being listed as “pay-to-participate” on ClinicalTrials.gov.

“Many of these businesses use websites, social media, YouTube videos, webinars and other tools to engage in direct-to-consumer marketing of supposed stem cell therapies. To my surprise, at one point I noticed that some of these companies had successfully listed “pay-to-participate” studies on ClinicalTrials.gov. Many of these “studies” look to me like little more than marketing exercises, though of course the businesses listing them would presumably argue that they are genuine clinical studies.”

While FDA-approved trials can charge study participants, most don’t. If they do, it’s motivated by recovering costs rather than making a profit. Turner also explained that organizations with FDA-approved studies “need to prepare a detailed rationale and a budget, and obtain approval from the FDA.”

Companies with unproven stem cell therapies are ignoring these regulatory requirements and listing their studies as “patient-funded” or “patient-sponsored”. Turner found seven such “pay-to-participate” studies sponsored by US companies on ClinicalTrials.gov. He also identified 11 studies where companies don’t indicate that patients have to pay, but do charge patients to participate in the studies.

Turner is concerned that these companies are using ClinicalTrials.gov to take advantage of innocent patients who don’t realize that these unproven treatments aren’t backed by solid scientific research.

“Patients have already been lured to stem cell clinics that use ClinicalTrials.gov to market unproven stem cell interventions. Furthermore, some patients have been injured after undergoing stem cell procedures at such businesses. Many individuals use ClinicalTrials.gov to find legitimate, well-designed, and carefully conducted clinical trials. They are at risk of being misled by study listings that lend an air of legitimacy and credibility to clinics promoting unproven and unlicensed stem cell interventions.”

Having identified the problem, Turner is now advocating for a solution.

“ClinicalTrials.gov needs to raise the bar and perform a proper review of studies before they are registered. Better screening is needed before more patients and research subjects are harmed. It’s astonishing that officials at the NIH and US FDA haven’t already done something to address this obvious matter of patient safety. Putting a disclaimer on the website isn’t sufficient.”

The disclaimer that Turner is referring to is a statement on the ClinicalTrials.gov website that says, “Listing of a study on this site does not reflect endorsement by the National Institutes of Health (NIH).”

Turner argues that this disclaimer “simply isn’t sufficient.”

“Patients and their loved ones, physicians, researchers, journalists, and many other individuals all use ClinicalTrials.gov because they regard the registry and database as a source of meaningful, credible information about clinical studies. I suspect most individuals would be shocked at how easy it is to register on ClinicalTrials.gov studies that have obvious methodological problems, do not appear to comply with applicable federal regulations or have glaring ethical shortcomings.”

While Turner acknowledges that the NIH database of clinical trials is a “terrific public resource” that he himself has used, he regards it “as a collective good that needs to be protected from parties willing to misuse and abuse it.” His hope is that his article will give journalists the starting material to conduct further investigators into these pay-to-participate studies and the companies behind them. He also hopes that “such coverage will help convince NIH officials that they have a crucial role to play in making ClinicalTrials.gov a resource people can turn to for information about credible clinical trials rather than allowing it to become a database corrupted and devalued by highly problematic studies.”

Convincing is one thing, but implementing change is another. Turner said in his interview that he knows that “careful screening by NIH officials will require more resources, and I am making this argument at a time when much of the political discourse in the U.S. is about cutting funding for the CDC, FDA, NIH and other federal agencies.”

He remains hopeful however and concluded that “perhaps there are ways to jolt into action people who are in positions of power and who can act to help prevent the spread of misinformation, bad science, and marketing packaged as clinical research.”

Texas tries to go it alone in offering unproven stem cell therapies to patients

/ Kevin McCormack / 5 Comments

Texas Capitol. (Shutterstock)

One of the most hotly debated topics in stem cell research is whether patients should be able to have easier access to unproven therapies using their own stem cells, at their own risk, and their own cost. It’s a debate that is dividing patients and physicians, researchers and lawmakers.

In California, a bill working its way through the state legislature wants to have warning signs posted in clinics offering unproven stem cell therapies, letting patients know they are potentially putting themselves at risk.

Texas is taking a very different approach. A series of bills under consideration would make it easier for clinics to offer unproven treatments; make it easier for patients with chronic illnesses to use the “right to try” law to take part in early-stage clinical trials (in the past, it was only patients with a terminal illness who could do that); and allow these clinics to charge patients for these unproven stem cell therapies.

Not surprisingly, the Texas bills are attracting some widely divergent views. Many stem cell researchers and some patient advocates are opposed to them, saying they prey on the needs of vulnerable people, offering them treatments – often costing thousands, even tens of thousands of dollars – that have little or no chance of success.

In an article on STATnews, Sean Morrison, a stem cell researcher at the University of Texas Southwestern Medical Center, in Dallas, said the Texas bills would be bad for patients:

“When patients get desperate, they have a capacity to suspend disbelief. When offered the opportunity of a therapy they believe in, even without data and if the chances of benefit are low, they’ll fight for access to that therapy. The problem is there are fraudulent stem cell clinics that have sprung up to exploit that.”

Patients like Jennifer Ziegler disagree with that completely. Ziegler has multiple sclerosis and has undergone three separate stem cell treatments – two in the US and one in Panama – to help treat her condition. She is also a founding member of Patients For Stem Cells (PFSC):

Jennifer Ziegler

“PFSC does not believe our cells are drugs. We consider the lack of access to adult stem cells an overreach by the federal government into our medical freedoms. My cells are not mass produced, and they do not cross state lines. An adult stem cell treatment is a medical procedure, between me, a fully educated patient, and my fully competent doctor.”

The issue is further complicated because the US Food and Drug Administration (FDA) – which has regulatory authority over stem cell treatments – considers the kinds of therapies these clinics offer to be a technical violation of the law. So even if Texas passes these three bills, they could still be in violation of federal law. However, a recent study in Cell Stem Cell showed that there are some 570 clinics around the US offering these unproven therapies, and to date the FDA has shown little inclination to enforce the law and shut those clinics down.

UC Davis stem cell researcher – and CIRM grantee – Paul Knoepfler is one of the co-authors of the study detailing how many clinics there are in the US. On his blog – The Niche – he recently expressed grave concerns about the Texas bills:

Paul Knoepfler

“The Texas Legislature is considering three risky bills that would give free rein to stem cell clinics to profit big time off of patients by selling unproven and unapproved “stem cell treatments” that have little if any science behind them. I call one of these bills “Right to Profit” for clinics, which if these became law could get millions from vulnerable patients and potentially block patient rights.”

Ziegler counters that patients have the right to try and save their own lives, saying if the Texas bills pass: “chronically ill, no option patients in the US, will have the opportunity to seek treatment without having to leave the country.”

It’s a debate we are all too familiar with at CIRM. Every day we get emails and phone calls from people asking for help in finding a treatment, for them or a loved one, suffering from a life-threatening or life-altering disease or disorder. It’s incredibly difficult having to tell them there is nothing that would help them currently being tested in a clinical trial.

Inevitably they ask about treatments they have seen online, offered by clinics using the patient’s own stem cells to treat them. At that point, it is no longer an academic debate about proven or unproven therapies, it has become personal; one person asking another for help, to find something, anything, to save their life.

Barring a dramatic change of policy at the FDA. these clinics are not going to go away. Nor will the need of patients who have run out of options and are willing to try anything to ease their pain or delay death. We need to find another way, one that brings these clinics into the fold and makes the treatments they offer part of the clinical trial process.

There are no easy answers, no simple solutions. But standing on either side of the divide, saying those on the other side are either “heartless” or “foolish” serves no one, helps no one. We need to figure out another way.