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 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.
By posting a comment, you agree to our terms and conditions.