Without rigorous evidence, claims are circulating freely that patients can benefit from “regenerative” therapies for a staggering array of conditions, including autism, aging, macular degeneration, Lou Gehrig’s disease, Parkinson’s, multiple sclerosis, lupus, and erectile dysfunction.
Patients are flocking to “clinics,” paying from $5,000 to as much as $50,000 a pop, to have fat, blood, and other tissues extracted from their bodies, processed and preserved in ways they likely don’t fully grasp, then re-introduced (injected) into areas of their bodies that give them pain or other problems.
A onetime corporate lawyer, debilitated by a stroke, was found to have a huge mass of primitive abnormal cells growing, tumor-like, at the base of his spine after he received multiple treatments at clinics in Mexico and Kazakhstan.
As jurors of the 2012 Nobel Prize in Medicine proclaimed, British biologist John B. Gurdon and Japanese physician Shinya Yamanaka “discovered that mature, specialized cells can be reprogrammed to become immature cells capable of developing into all tissues of the body. Their findings have revolutionized our understanding of how cells and organisms develop.”
The prize citation went on to note that the scientists’ “groundbreaking discoveries have completely changed our view of the development and cellular specialization. We now understand that the mature cell does not have to be confined forever to its specialized state. Textbooks have been rewritten and new research fields have been established. By reprogramming human cells, scientists have created new opportunities to study diseases and develop methods for diagnosis and therapy.”
Over time, researchers have advanced, simplified, and spread the alternative use of “induced pluripotent stem cells,” allowing for serious study of conditions that might be treated with materials (such as stem cells) from patients’ own bodies and further advancing regenerative medicine. It’s a field that some trace to ancient Greeks’ observations that the body can repair itself and regrow key components as some lizards do with missing limbs and tails.
But the rigorous study that was supposed to occur in major, careful institutions didn’t always occur exactly that way. Stem cells and regenerative medicine are important, promising areas for medical advancement (see below). They also quickly became areas for exploitation, first overseas, then on American shores.
But if doctors in the Western developed world had umbrage for unproven stem cell treatments in the East, that fast fell with the rapid spread of such practices into places where regulation and oversight supposedly was tougher.
Basketball superstar Kobe Bryant stunned the sports world when it became public that he had traveled to Germany to receive platelet-rich plasma (PRP) therapy. It’s a treatment in which patients’ blood is extracted, and then spun down so it is concentrated with purportedly healing platelets and stem cells. This viscous material is injected into joints, supposedly helping them repair themselves and reducing the wear and tear of rheumatoid arthritis, age, and professional play. Bryant recommended PRP to baseball star Alex Rodriguez, who was outspoken about how he said it helped him with his bad knee.
Further, they wrote: “U.S. businesses promoting stem cell interventions claim to treat a wide range of diseases and injuries, as well as advertising stem cells for cosmetic applications, ‘anti-aging,’ and other [niche] purposes … such procedures as ‘stem cell facelifts’ and ‘stem cell breast augmentation,’ as well as sexual enhancement procedures. Orthopedic and sports medicine clinics often promote stem cell interventions for joints and soft-tissue injuries. Other clinics … list stem cell interventions for 30 or more diseases and injuries [including ] neurological disorders and other degenerative conditions, spinal cord injuries, immunological conditions, cardiac diseases, pulmonary disorders, ophthalmological diseases and injuries, and urological diseases, as well as cosmetic indications.”
The scientific duo said the proliferating practices raised troubling medical, ethical, and regulatory issues, not only because they offered little or no evidence for their use or outcomes but also because the targets for the medical services included parents or their underage kids with health conditions or diseases, including autism, cerebral palsy, and muscular dystrophy.
That’s because those hustling commercial stem cell care, sometimes in vanilla office buildings or store fronts and outside of well-known academic medical centers and top-line hospitals, have managed to tap dance on a fine legal line about stem cell and regenerative treatments.
The FDA, as Gottlieb notes, sees a gray area when providers take blood, fat, or other cells from patients and do little to the materials before reintroducing them into their bodies. The more that they process, add, or otherwise alter the cells, the higher their regulatory exposure.
But as Gottlieb has written, when materials are “taken from and given back to the same individual or when the cells or tissues do not undergo significant manufacturing, are intended to perform the same basic functions, and are not combined with another drug or device, among other factors; their benefits and risks are well understood.”
The field changes rapidly, however, as do the promises and downright scams.
Regenerative medicine holds great promise still. But not for patients right now.
It would be great if medical science made progress easily and quickly. It just doesn’t happen that way. Experts estimate it typically can take almost two decades for clinicians to fully and safely adopt a medical advance.
Still, for all the attention devoted to stem cells and regenerative medicine, careful and precise medical scientists talk mostly about the numerous investigations under way, and how indications are still solid that various lines of research will prove beneficial. They do not boast about “cures” or “breakthroughs” or mostly proffer purely anecdotal support for their research outcomes.
The society insists that safe and effective stem cell and regenerative treatments can best be advanced through stringent science, including rigorous clinical trials that can take years to conduct correctly. Such medical research may seem pokey and cumbersome to non-scientists. But its emphases on transparency and results that are fully documented (with hard data and scientific evidence) and can be repeated are invaluable. So, too, are the many safeguards built into these time-tested processes. This includes researcher oversight, by Institutional Review Boards and external regulators, and robust, careful communication about human experimentation: Clinical trial participants must know fully what will and may happen to them when they are subjected to tests or procedures. This informed consent is a critical, fundamental part of safe, ethical studies involving human subjects.
It’s troubling to note that the NIH is under some fire now for failing to closely monitor its vital, central database on clinical trials, with some direct-to-consumer purveyors of purported stem cell treatments getting onto this list (www.clinicaltrials.gov) in what one researcher has termed “pay to participate” ploys. As a result of his publication criticizing non-FDA approved testing getting on the clinical trials list, bioethicist LeighTurner has received legal threats. So now there may be dual, unacceptable challenges to stringent medical science from those claiming to offer stem cell care: Will they undercut the deep, hard-earned respect that careful clinical trials have won, and will they stifle appropriate, scientific publications questioning how the (in this case, tax-supported) medical establishment operates?
Why skepticism is smart
Caveat emptor applies to faddish stem cell and regenerative therapies.
In my practice, I see the significant harms that patients suffer while seeking medical services, and I can’t see any good reasons for most Americans to even consider stem cell or “regenerative” treatments, especially as long as they’re not FDA-approved and offered by folks who can’t or won’t provide detailed data as to their outcomes and benefit. The FDA, doctors, licensing authorities and others must do more to crack down on providers promising, without scientific evidence, stem cell or regenerative treatments for myriad diseases and conditions.
Anecdotes aren’t evidence. Patient stories can be powerful, but they shouldn’t be decisive for your health choices. Doctors can give some patients sugar pills and saline (sterilized salt water) injections, and they will report improvements in their condition. That’s because of the placebo effect, which is well documented and can play a role in patients’ sense of a treatment’s effectiveness (see below). But even where they may be evidence-based regenerative and stem cell treatments, such as cord blood banking, public health officials must ensure claims for them don’t get exaggerated and their use out of hand.
There are, however, less parlous ways than “regenerative” treatments to feel better. Let’s be clear: Every medical service carries a risk. Providers may insist that jabbing you, repeatedly, in the arm for blood or the hip for fat or marrow causes little discomfort, particularly because they’ll give local anesthesia. But how certain will you be about your safety from infection, including for hepatitis or even HIV-AIDs? Or that the extraction needle will go exactly where it is supposed to without other harm? When your blood or fat or marrow has been harvested, how can you be sure it will be handled in sterile, appropriate ways? Or will you get back, as one mother did, a supposedly regenerative product—in this case, her processed placenta from her recent delivery— tainted with strep?
While you’re serving as a human guinea pig for unproven stem cell approaches, are you harming yourself by delaying more appropriate care? Growing research, for example, suggests that patients and orthopedists may be taking a less than optimal path with minor joint procedures like “scoping” of knees, hips, and shoulders, or injecting them with steroids or natural body lubricants. They may benefit patients more, instead, by determining their joints are sufficiently damaged, unlikely to function better, and to replace them. By the way, when you’re researching purported regenerative joint care, don’t ask only your orthopedist but maybe also consult your family doctor, internist, or a rheumatologist. Orthopedists are, by practice, action-oriented specialists, and, as mentioned, research shows their inclination to injections (of steroids or lubricating substances) isn’t always the prime course.
Though Vanity Fair tries to present some balance and notes in its coverage the absence of scientific support or FDA approval for stem cell therapies, the magazine also offers this flat statement that the film “offers a persuasive side of the story, with rapturous testimonials from patients, some of whom who have regained the ability to walk after their stem cell vacations. Added bonus: They come home with better skin, bigger sex drive, and (in the case of at least one balding patient) more hair.”
Why should we care? As the California agency charged with boosting stem cells and regenerative medicine in rigorous and beneficial fashion has observed: “The bulk of the danger with [celebrity] stories like these is not that the individuals involved are likely to be harmed (though they may be) but that the attention their famous names draw to stem cell therapies could lead others to copy their example, and undergo procedures for which there is no evidence to show they are safe and effective.”
This is a good thing, because celebrities’ power to do good—by raising health awareness or public or financial support—must be balanced by their possible harms, including popularizing risky therapies. Skepticism and moderation matter where our health is concerned.
A caution about the placebo effect
The mind can exercise great sway over the body, and patients often report reactions—positive and negative— to medical treatments based not on measurable evidence but on hope and belief.
Doctors long have known that some patients respond simply to the attention they get, not necessarily to a given medical service. They have found they can get good results just by taking the time to chat with patients and then by giving some—not just hypochondriacs or those too tightly wired—a sugar pill or a shot of saline.
To be sure, the placebo effect isn’t typically credited with reversing a disease or condition. It’s unlikely to move the metrics for fever, blood cell counts, or other body markers. But it can buoy patients’ spirits, reduce their perception of problems like pain, and make them feel better.
Placebo effects can pose big woes for serious medical science, which tries to sort out biological from psychological effects by conducting “double-blind” studies. That means that neither clinicians nor participants in a study on a given medication will know who got an experimental drug or service and who got the sugar pill.
Because providers of untested stem cell and regenerative treatments haven’t subjected their approaches to rigorous scientific studies, it’s unknown whether patients feel better after getting them because they work or due to the placebo effect. Researchers speculate that the latter may be truer.