An Insider’s Antidote for Dangerous Stem Cell Hype

Xconomy San Francisco — 

UC Davis professor Paul Knoepfler’s new book about stem cells may never break into the New York Times best-seller ranks, but he still might make some publishing history.

Knoepfler could be the only biology researcher ever to write a comprehensive consumer guide about the health care treatments being developed in his own field—complete with safety cautions.

Stem Cells: An Insider’s Guide is also a scientific primer and a paean to the promise of stem cell research. But Knoepfler, whose 370-page book originated as a blog about his field, also wanted to protect the thousands of vulnerable patients who have prematurely looked to stem cell therapy as a last-ditch hope for a cure.

His greatest concern is for patients who have been lured by the false claims of dubious for-profit clinics to pay as much as $100,000 for unproven “stem cell treatments.” These injections, he says, may actually consist of cells culled from pigs, cows, or sheep, and bear unknown risks such as contamination or immune system reactions.

A growing network of secret online communities, as well as overt advertisements, have spurred US patients to pay fortunes out of pocket to ill-trained physicians, who perform experimental procedures backed by little or no evidence, says Knoepfler, an associate professor and researcher at the UC Davis School of Medicine’s Institute for Regenerative Cures.

“The number of patients being treated, and the number of providers, are increasing,” Knoepfler says. “I don’t think the FDA has the budget to deal with this.”

But Knoepfler’s book is also aimed at patients who are eager to enter FDA-approved clinical trials, confident that stem cell treatments will soon deliver miracle cures with few downside risks.

“One of the motivations for my book is letting people in on some of the realities,” Knoepfler says.

Knoepfler's book is published by World Scientific.

Knoepfler’s book is published by World Scientific.

Knoepfler tells patients what many of them don’t want to hear—that very few stem cell therapies have yet been proven safe and effective, although thousands of clinical trials are under way. What’s more, testing those therapies requires even more careful safety monitoring than many other kinds of treatments, he says.

Stem cells pose unique risks because they are alive and can in theory grow inside the body, which makes them very different from most chemical or biologic drugs, Knoepfler says. Patients who have a bad reaction to a drug can stop taking it, and the body will eliminate it, he says.

“If you get injected with a billion living cells, the truth is, we really don’t know what happens after that,” Knoepfler says. One of the possibilities recognized by researchers is that the stem cells may eventually initiate the growth of a tumor—an issue that Knoepfler explores in his own lab at UC Davis.

That said, Knoepfler thinks the promise of stem cells will prove out, given enough time to carefully work out the bugs.

“I’m very optimistic about the stem cell field,” he says. “I think it will transform medicine in the coming decades.”

In his book, Knoepfler starts out with a friendly tour of the basics about stem cells. While most people know something about embryonic stem cells, which are derived from very early stage embryos, stem cells also occur naturally in many bodily tissues. Stem cells called “induced pluripotent cells” have also been created in the lab by manipulating adult skin cells. Stem cells retain the ability to morph into many kinds of specialized cells—a trait that has lead to the excitement over their potential as disease treatments. Theoretically, stem cells, or specialized cells derived from them in a lab, could be used to replace damaged tissues such as nerves or heart muscle.

But as Knoepfler emphasizes, scientists still don’t know how a stem cell might change in the body decades after being injected. He cites the case of a woman who got a “stem cell facelift,” and later suffered from a bone growing in her eye.

He doesn’t skirt other challenges facing developers of legitimate stem cell therapies. To inject a billion cells, for example, researchers must induce the desired stem cells to grow into larger populations in the lab. But in the course of multiplying, some of the cells may lose desirable traits, or develop abnormal features.

While many of Knoepfler’s chapters describe such harsh realities, the book often shares the lighthearted tone that had earlier emerged in his blog as he tried to engage patients, fellow scientists, and even the operators of unlicensed “stem cell” clinics in a dialogue about the fledgling field. The book is sprinkled with quips, Knoepfler’s own opinions, and his hand-drawn cartoons.

Knoepfler tackles questions and rumors that would never make the agendas of scientific meetings: “Are there stem cells in Pepsi?” and  “Is there a secret government plot against stem cell therapies?”

As a university professor, Knoepfler had hesitated when he first started thinking about launching a blog in 2009. “Academic scientists are taught to stick to more scientific forms of communication, like papers and meetings,” he says.

But when Knoepfler looked at the social media environment, he saw a need for an accessible public platform for well-founded scientific evidence and debate. A popular Nature blog on stem cells, The Niche, had been discontinued. Knoepfler found the searchable online universe dominated by opponents of stem cell research on one side, and seemingly shady providers touting spurious stem cell treatments on the other.

Knoepfler’s doubts about writing an informal blog for the public faded in November of 2009, when he was diagnosed, at age 42, with an aggressive form of prostate cancer.

“It made me less afraid of challenging the expectations of academic scientists,” he says.  Knoepfler says he felt an increased obligation to help other patients who were searching for reliable information, while feeling they were running out of time. Knoepfler’s own cancer is now in remission following treatment.

His blog, launched in 2010, now draws about 3,000 readers a day. It also attracted interest from publishers, including World Scientific Publishing of Singapore, which released Knoepfler’s book through Amazon in late September.

Although Knoepfler isn’t an MD, he receives queries about once a week now from patients seeking his advice on their own medical issues. He tries to gently steer them away from unlicensed providers. But often, they find it hard to abandon their faith in stem cells when few alternatives are available, he says.

“They think, ‘This is going to help us, whereas other medicines have failed,’ ” Knoepfler says.  Most disturbing, he says, is the fact that parents are agreeing to have their children undergo unproven stem cell treatments for disorders such as autism—a complex, poorly understood condition not verifiably linked to any specific damaged cell that might be replaced by stem cell therapies.

Surprisingly, Knoepfler also trades emails with treatment providers who don’t comply with FDA regulations—a significant portion of his readership. He tries to persuade them to follow the law and avoid arrest.

“They send me information about their less worthy competitors,” Knoepfler says wryly. But Knoepfler, as well as the International Society for Stem Cell Research, has also been threatened with lawsuits for taking a tough public stance on non-compliant providers.

Knoepfler’s concerns are shared by research-related institutions. A group of 13 professional organizations, including the International Society for Stem Cell Research and California’s stem cell funding agency, the California Institute for Regenerative Medicine, issued a joint statement Aug. 19 warning consumers to be wary of clinics making false promises about stem cell treatments while failing to observe US regulations or guidelines in other countries where they operate. The FDA has issued similar consumer advisories.

In addition to protecting individual patients, Knoepfler says, these warnings may forestall a catastrophe that could delay progress in stem cell research, just as the deaths of clinical trial subjects in gene therapy trials in the late 1990’s froze research in that area.

“If something profoundly bad goes wrong, it could affect the whole field,” Knoepfler says.

As for Knoepfler, he hasn’t detected any career setbacks due to his frank blog posts about the challenges in his field. One fellow researcher from another university asked him what his department chair, his dean, and their lawyers said when he told them he was about to launch the blog. Knoepfler says he had never sought their go-ahead.

“I don’t know what would have happened if I had actually asked for permission,” Knoepfler says.


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8 responses to “An Insider’s Antidote for Dangerous Stem Cell Hype”

  1. Alberto Salazar says:

    Once again, a researcher who feels safe to side with the FDA contrary to those
    researchers apprehensive to contradict Dr. Knoepfler’s claim that the FDA is
    not right to claim that autologous stem cells from a patients own body is a
    drug but a medical procedure, the repercussions of losing funding when contradicting
    the FDA is too much of risk. We can have thousands of riskier procedures like
    heart transplants, bone marrow transfusions, etc. as medical procedures and
    adhere to medical state boards, but Dr. Knoepfler along with those that side
    with the FDA have captured a medical procedure in an unrealistic realm of
    expensive FDA drug clinical trials believe it belongs in this paradigm which
    does not. Meanwhile, thousands of patients wait in the sidelines ill. Dr.
    Knoepfler’s constant campaign to defend the FDA is doing a total disservice to
    the ill and terminally ill.

  2. HeraSentMe says:

    PK is certainly no hero, no patient advocate, etc. He posts his self-aggrandizing, megalomaniacal, tabloid style blog posts purely for shameless self-promotion. Here is one example of a REAL patient advocate:

  3. HeraSentMe says:

    See Dr. Chris Centeno for someone truly worthy of praise!

  4. HeraSentMe says:

    All the members of Patients For Stem Cells and Stem Cell Pioneers are the ones fighting daily, not only for their lives, but also, for the lives of others… while PK posts absurd tales worthy only of the Enquirer! And as PK is so busy feeding his ego, he seldom publishes any research papers. Where does all that CIRM, taxpayer funded $$$ go?

  5. HeraSentMe says:

    Ms. Tansey, you should do your homework before praising a fraud like PK.

  6. Zchick1836 says:

    As an adult stem cell patient for MS the United States lags way behind in biologics. A patients own stem cells taken from their own body have been proven safe with thousands and thousands of patients. Those who aren’t on board with the idea of a patient being treated with their own stem cell for disease/conditions have many reasons why….Research $$$’s to their universities would dry up, book deals would disappear, speaking engagements would vanish, ego’s would be bruised, jobs would be at stake. Some scientists have put all their eggs in one basket. If a scientist could create a bio-equivalent product or a generic, say an iPC cell, then it could be sold to the public as a drug marked up x100. This would make the FDA happy, the pharmaceutical companies quite happy but leaves “NO OPTION PATIENTS” hung out to dry in the “valley of death” waiting at a chance for quality of life improvements. As a friend of mine, SammyJo Wilkinson, who is also a very public activist for adult stem cell treatment said best…”Paul, while you’ve got your head in the clouds, (referring to an article that PK wrote) physicians are down in the trenches everyday with suffering patients. We support Dr. Centeno an true advocate for patients because he recognized, then documented the higher efficacy stem cells offer to patients. Then he stood up to the overzealous regulators. We’ve had the safety argument with you over the past year and a half, autologous MSCs, even expanded, continue to mount a strong safety profile as in Centeno’s reports, and in University phase 1/2 clinical trials. It’s time to bridge this divide. Join us in the call to allow physicians to to practice medicine with expanded cells. Our own stem cells are like homemade clothes. There will be time for designer ipsc’s, but what MSCs do naturally should be fully examined and understood first. Patients treated with their own cells should be the benchmark against which man-made cell therapies are measured.” Anyone can call them self a “patient adcovate,” but ask patients who we think the true patients advocate are, and you’ll hear names like Dr. Chris Centeno, Dr. Neil Roirdan, Dr. Doug Broeska and Dr. Camillo Ricardo. Adult stem cell patients have bonded together and formedhttp://www.patientsforstemcell/… We feel our civil rights have been violated!

  7. Zchick1836 says:

    Why would you not post my comments when they were stated politely? I’m just of a different opinion and that’s not allowed? You’ve left some other nasty comments up but taken mine down? WOW! Please be a fair and unbiassed journalist. Thanks! Jennifer Ziegler