Legit Stem Cell Therapies Advance as Bogus Clinics Proliferate

Nancy A. Melville

June 11, 2018

NASHVILLE, Tennessee — Stem cell transplantation research continues to show encouraging benefits in inducing long-term remission of multiple sclerosis (MS), but many are cashing in on the hopes raised by headlines of "game-changing" results, to the detriment of patients as well as the research field in general, experts say.

"This is an issue that is obviously of a lot of interest to our patients, and there are places cropping up that are taking advantage of that, offering stem cell therapy and feeding off of a vulnerable population that is desperate for anything," Mark S. Freedman, MD, a professor of medicine and neurology at the University of Ottawa and director of the Multiple Sclerosis Research Unit, Neurology at Ottawa Hospital-General Campus, in Ontario, Canada, told reporters at a press conference here at the Consortium of Multiple Sclerosis Centers (CMSC) 2018 Annual Meeting.

Driving the high interest in stem cell transplantation for MS are ongoing clinical trials showing results that are indeed impressive, summed up in a recent meta-analysis  of 15 studies and 764 transplanted patients.

Published in 2017 and reflecting research from 1995 to 2016, the analysis showed as many as 83% of patients with relapsing-remitting MS treated with hematopoietic stem cell transplantation (HSCT) maintained no evidence of disease activity (NEDA) at 2 years, and 67% at 5 years, representing significantly higher rates than those seen even with the most effective MS therapies.

Treatment-related mortality was meanwhile just 2.1%, with higher rates seen in the earlier studies and among patients with higher levels of disability.

In Freedman's own research in Canada, further encouraging results were reported, with 23 of 24 patients with relapsing-remitting MS remaining relapse-free as long as 13 years after an autologous HSCT.

However, the HSCT treatment being offered in Canada but not approved by the US Food and Drug Administration (FDA) for MS, is a far cry from the wide variety of unapproved and usually ineffective processes being promoted by direct-to-consumer stem cell clinics as treatments for everything from MS to weight loss, heart failure, and even autism.

Scenarios reported at such clinics may involve the removal of adipose cells from somewhere such as the abdomen, processing of the cells with a variety of unproven methods, and then replacement of the cells, with likely little to no improvement to the patient — after costing them thousands, reportedly anywhere from $5000 to $50,000.

As Freedman explains, a legitimate stem cell transplant for MS would involve a vastly more complicated set of procedures, including carefully regulated conditioning of cells, while patients meanwhile undergo arduous immunoablation with chemotherapy to wipe out most of their immune cells and prepare for a reprogramming of their system with the new cells.

"You could certainly get some stem cells from fat the way [the clinics] do it, but only a tiny amount — we are getting one to two million cells per kilo, so you need billions of cells and you're not going to get that from one extract," he said.

"The cells are then purified and grown in highly regulated, certified conditions to prevent contamination. It's just ludicrous to think this could be done in the same day (as some clinics advertise)," he said.

Reports of potential harms to patients at clinics providing unproven stem cell treatments range from infections and complications to deaths — and then there is the additional loss of thousands of dollars for an ineffective therapy.

Also important is the toll taken on the field in general, with a deterioration of public trust in efforts to advance the legitimate field of stem cell research, Freedman told Medscape Medical News.

"When patients get bogus treatments and they don't work, they start to question why we are doing all this work for nothing," he said. "Some may have influence on the funding of the field."

In discussing potentially bogus stem cell interventions with patients with MS, Freedman noted that patient desperation in finding effective treatment — combined with the hype of unproven therapies —  may prove to be a barrier.

"I think that we do what we can, but patients are not always opening up to us when they go shopping elsewhere for their care," he said.

"I agree that clinicians should take more of a stand [on direct-to-consumer stem cell clinics] and inform patients about the risks, but patients may not believe we are telling them the whole story."

"Scienceploitation"

In a talk at the CMSC meeting addressing the issue of controversial direct-to-consumer stem cell intervention clinics, legal expert Timothy Caulfield noted that one of the most significant factors driving what he calls "scienceploitation," the overhyping of unproven stem cell interventions, has been the scientific community itself, which, faced with controversy from the start, was backed into a corner of needing to heavily promote the benefits of the research.

"There were so many problems right out of the gate when this started," Caulfield, who is the Canada Research Chair in Health Law and Policy and professor in the Faculty of Law and School of Public Health at the University of Alberta, Edmonton, Canada, told a packed conference room at the meeting.

"The scientific community had to make big promises to counter the opponents, so almost immediately you had a lot of hyperbole about the harms as well as the benefits."

Caulfield and his colleagues conducted an analysis looking at how the timeline for the expected arrival of the research into the clinic was being presented in the media, and found that nearly 70% of reports stated the process would be available in the clinic within 5 to 10 years or sooner — further driving public excitement and demand.

"This creates an expectation in the public that can be exploited by these stem cell clinics," he said.

Most reports in the popular press furthermore were positive or neutral regarding the unproven therapies, focusing on anecdotal reports of "miracle" cures, with little emphasis on the risks of the unproven therapies, thereby driving more demand.

And in some cases, media reports even provided information on how the public could help fund or crowdsource patients' travels to countries where they could receive the treatments.

Whereas unproven stem cell interventions were once primarily offered in only a few countries outside of the United States, as many as 570 direct-to-consumer stem cell clinics were recently reported to be operating in the United States.

According to the report, the leading conditions being marketed are orthopedic treatments; pain; sports injuries; and neurologic,  immunologic, and respiratory conditions.

In comments to Medscape Medical News, Caulfield noted that patients with MS may be particularly vulnerable to the bogus treatment clinics.

"Many of these clinics target MS patients," he said. "It is a disease with only a few treatment options and stem cell science would seem, intuitively, to apply."

"Also, there is very promising real science happening in this space, and this work gets media exposure," he added. "I think this may make the unproven therapies seem more plausible." 

FDA, Regulators Cracking Down

As reported by Medscape Medical News, the FDA has stepped up efforts to crack down on the bogus stem cell clinics, recently seeking permanent injunctions against two clinics —  the California Stem Cell Treatment Center Inc, with locations in Rancho Mirage and Beverly Hills, California, and the US Stem Cell Clinic LLC of Sunrise, Florida — for marketing unapproved stem cell products and violating manufacturing requirements to ensure sterility of products.

California has meanwhile passed legislation mandating that stem cell clinics to explicitly note that their treatments are experimental and not FDA approved. Australia, which reportedly had among the world's highest concentration of direct-to-consumer stem cell clinics, now prohibits the clinics from advertising altogether, with fines of $4.2 million and $840,000 for advertisement of services for autologous human cells and tissue products or use colloquial terms such as "stem cells," respectively.  

Meanwhile, in its guidelines, the International Society for Stem Cell Research has called on clinicians to be proactive in informing patients of the risks presented by clinics offering unproven stem cell treatments.

"This is part of your duty as a scientist," Caulfield asserted. "To not hype this work, but also correct the record when you see something wrong."

He called on clinicians to "be part of the conversation — be part of the solution."

Freedman's disclosures include honoraria or consultation fees from Bayer Healthcare, Biogen-Idec, Chugai, EMD Canada, Genzyme, Novartis, Sanofi-Aventis, and Teva Canada Innovation. He is a member of advisory boards, board of directors or other similar group for Bayer Healthcare, Biogen-Idec, Hoffman La-Roche, Merck Serono, Novartis, Opexa, and Sanofi-Aventis. Freedman is a participant in Genzyme's company-sponsored speaker's bureau. Caulfield has disclosed no relevant financial relationships.

Consortium of Multiple Sclerosis Centers (CMSC) 2018 Annual Meeting. Presented June 1, 2018.

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