FDA moves to crack down on unproven stem cell therapies

LOS ANGELES — Federal regulators are preparing to crack down on scores of clinics across the United States that offer pricey stem cell therapies for conditions ranging from autism to multiple sclerosis to erectile dysfunction without any scientific evidence that they work.

As many as 200 stem cell clinics have cropped up in recent years, peddling injections, facelifts, and treatments for a number of devastating conditions. They have avoided heavy regulation, in part because they use cells extracted from a patient’s own body and because they don’t do much to those cells before reinjecting them.

But the Food and Drug Administration recently issued draft guidelines clarifying that the stem cells used in most clinics are drugs and require a rigorous approval process before they can be used in patients. A public hearing is set for April.

The FDA underscored the new approach in a warning letter it sent at the end of December to a network of stem cell clinics in California, New York, and Florida. Regulators advised the owner that he needed FDA licenses and approval to sell and use stem cells, which the agency classified in the letter as biological drugs. Such licenses would require evidence that stem cell treatments are both safe and effective — the sort of proof that takes drug companies many years of clinical trials to obtain, at a cost of millions of dollars.

Anxious patients plan to flood the public hearing, scheduled for April 13 in Silver Spring, Md., and assert their rights to use their own cells as they see fit.

The FDA’s moves come after years of pressure from physicians and researchers who have called for a crackdown on an unproved therapy that they consider dangerous quackery. These critics say there’s no evidence the treatments work — or that some of them even contain stem cells. Yet clinics charge fees ranging from $5,000 to $25,000 per treatment, with some patients reportedly racking up bills over $100,000.

While there haven’t been many reports of serious complications from stem cell therapy, two Florida patients died in recent years after receiving stem cell injections; a California woman developed painful bone fragments in her eyelids after a stem cell facelift; and another patient developed a mucous-secreting growth of nasal tissue in her spine after undergoing stem cell treatment in an attempt to cure her paralysis.

“It’s a huge, unapproved human experiment,” said Paul Knoepfler, a stem cell researcher at the University of California at Davis who tracks for-profit stem cell clinics on his blog “The Niche.”

The FDA did issue a public warning about stem cells in 2012 and has sent a series of warning letters to individual clinics in recent years, mostly involving issues of sterility and disease prevention. In some cases, regulators warned that the processes used by the clinics turned the cells into what the FDA would consider drugs. A handful of clinics targeted by the agency have moved their operations out of the US.

But that’s not the norm: Patients don’t have to travel to Mexico or India to get stem cell therapy, as they would for many other questionable treatments. There are clinics even in rural states like Kansas and Nebraska.

Leigh Turner, a bioethicist at the University of Minnesota who has been pressing the FDA for years to crack down on the clinics, said he has been amazed that regulators have allowed the industry to grow so rapidly.

“If it’s not safe and it’s not going to help patients,” Turner said, “it’s just predatory behavior.”

Brushing off charges of ‘quackery’

Dr. Mark Berman has heard such criticism often.

“Doctors say it’s quackery, nonsensical, no better than placebo,” Berman said. “Everyone on the planet thinks I’m a charlatan and we’re just in it for the money.”

An entrepreneurial cosmetic surgeon based here in Beverly Hills, Berman has been providing stem cell treatments since 2010 and cofounded a network of clinics in 2012. He uses liposuction to extract fat from the patient, then spins it in a centrifuge with various enzymes for about a half-hour to separate a host of cells, including stem cells. He says he uses lab tests to verify that stem cells are present.

He then injects this “soup,” as he calls it, back into patients. Berman charges $8,900 per treatment (though he sometimes gives free or discounted care to financially strapped patients). He says the injections have been most successful for orthopedic issues, arthritis, and joint pain.

But a website for his Cell Surgical Network, an umbrella for dozens of stem cell clinics nationwide, lists more than two dozen other conditions the physicians are “currently studying,” including Parkinson’s, amyotrophic lateral sclerosis — more commonly called Lou Gehrig’s disease — congestive heart failure, lung disease, glaucoma, and muscular dystrophy.

The website is careful not to promise that the stem cell injections can cure or treat those diseases, and Berman said he makes it clear to all patients that the work is investigative and not FDA-approved.

Berman acknowledges that he has no published studies to back up his treatment. But he says he’s certain it works and is safe. As proof of his confidence, he notes that he used the therapy to successfully treat his wife for hip pain.

He says critics, including pharmaceutical companies and academics, want to profit by patenting stem cells and fear “disruptive technologies” that come from entrepreneurs rather than from their own incremental research.

Berman plans to attend the FDA hearing to argue, as other clinics have, that the injections are not drugs, but simple outpatient surgeries that should not be regulated.

Real promise, vastly overhyped

There are two main types of stem cells. Those derived from embryos can turn into almost any other type of cell in the body. Adult stem cells, specific to certain tissues, are not quite as flexible, but still serve as a kind of internal repair system.

Because of their remarkable regenerative powers, both embryonic and adult stem cells hold the promise of curing a variety of ills. Stem cells extracted from bone marrow have long been used to treat cancer and blood and immune disorders.

At least one published study shows stem cells extracted from fat tissue can speed healing of grafted tissue.

Clinical trial evidence also suggests the cells might speed wound healing, improve heart function, and treat scleroderma, said Dr. Peter Rubin, a stem cell researcher who chairs the department of plastic surgery at the University of Pittsburgh Medical Center. He is running a clinical trial to test the use of stem cells to repair severe facial wounds in soldiers.

But Rubin says any new treatments based on stem cells are years away.

And there’s no evidence yet that the cells can heal the variety of grave conditions, from stroke to incontinence to lung disease, that the clinics claim to treat — and no proof, other than the word of clinic operators, that they are harvesting the stem cells cleanly, or even harvesting them at all.

“What’s in the syringe? My guess is nothing helpful,” Knoepfler said. “It’s really scary to think there are thousands of people being injected with stuff, and in most cases we don’t know what that stuff is.”

The FDA is charged with regulating the human use of biological products, such as vaccines, blood, tissues, cells, and genes, and requires many of these items to gain approval as drugs.

But there are exceptions for biological products that are “minimally manipulated” and are taken from and put back into the same patient during a single surgical procedure, so they pose little risk of spreading infection. Stem cell clinic operators have long claimed their products fall under these exceptions.

And until now, the FDA has largely left them alone.

Given that history, some industry observers say it’s possible the latest regulatory push will fizzle. Others, however, believe the agency is ready to insist on higher standards that will force many clinics to shut down or move abroad.

A Kardashian endorsement

Unapproved stem cell therapies came into vogue about a decade ago and have exploded in popularity.

Stem cell lotions, some selling for hundreds of dollars per ounce, claim to reverse aging and erase wrinkles, though experts say there’s no reason to think stem cells or their extracts would remain active in a lotion — if, indeed, they were ever added to the jar.

“It could be stem cells,” said Rubin. “Or bacon grease.”

One step up from the lotions are $500 stem cell facials, which have quite a following among celebrities; Kim Kardashian gushed about the one she received before her 2014 wedding. Stem cell experts note that these treatments, which mainly use plant stem cells applied topically, are hardly likely to regenerate human skin.

Stem cell face-lifts are popular, too: Stem cells or stem cell extracts are injected into the skin along with fat. An American Society of Plastic Surgeons task force convened in 2012 and cochaired by Rubin found the procedures offered no improvement over standard facelifts.

Most troubling to many physicians are the stem cell clinics that market to patients desperate for relief from painful conditions or cures for terminal diseases.

“All of us hate seeing those big billboards for stem cell therapy,” said Rubin, who worries that problems with unproven stem cell therapies will taint the field. “The key is to move this forward with solid, well conducted studies.”

He added: “We need to be cautious of spurious, unjustified, and unsubstantiated claims.”