Stem Cell Treatment: What You Should Know

These days, there's no shortage of bold claims being made about the overwhelming array of conditions that can be treated by stem cell therapy. Search online and you can essentially find "a stem cell clinic somewhere in the world that will offer to cure almost anything under the sun, ranging from Alzheimer's ... to Parkinson's," points out Dr. Deepak Srivastava, president-elect of the International Society for Stem Cell Research and president of Gladstone Institutes, an independent biomedical research institution in San Francisco.

In the U.S alone, there are hundreds of stem cell treatment clinics offering therapies for a wide range of conditions. The problem? For all the buzz that has surrounded the supposed miracle potential of stem cells much more research is still needed.

What is Stem Cell Therapy?

Stem cells are special human cells that have the potential to develop into almost any type of cell. Most stem cells used in research or medicine today come from one of two sources. Some are human embryonic stem cells, derived from eggs fertilized in vitro (outside of the body) and donated for that purpose. This source of stem cells remains controversial and is opposed by some on moral grounds. The other source comes from adult stem cells, derived from fully developed tissue, like bone marrow.

Because they have the potential to develop into such a broad range of cells, stem cells can sometimes to be used to help in repair of tissue. Still, for all their inherent promise, the ways in which stem cells have been so far proven effective for medical use is far more limited. One primary way stem cells (in this case derived from bone marrow) are successfully used today is in helping to heal orthopedic injuries like bone fracture defects, where the bone isn't otherwise able to heal properly, and ligament or tendon injuries. Bone marrow transplants are also used for some cancer patients. Through this procedure, patients with leukemia and other cancers that affect the blood or immune system receive an infusion of healthy blood stem cells that allows them to receive higher doses of chemotherapy or radiation.

Outside that narrow scope of effective uses for stem cells, however, broad claims are being made by some stem cell treatment clinics that go far beyond what current science supports. Experts say those exaggerated claims are taking advantage of the most vulnerable and desperate. That includes patients and families desperately searching for cures modern medicine has yet to develop -- like a cure for autism, Alzheimer's disease, MS and Parkinson's disease. "The fact of the matter is that there simply have not been clinical trials that have demonstrated efficacy (of stem cell treatment) for any of those diseases as yet," Srivastava emphasizes.

The High Cost of Stem Cell Therapy

"I'm really concerned about all these for-profit stem cell clinics that are directly marketing to consumers," adds Paul Knoepfler, a stem cell biologist at the University of California at Davis. He's been an outspoken critic of the for-profit stem cell industry. "Because what they've done in large part is taken this massive shortcut around all that hard work that you really need to do to know what you're doing -- to know what you're working with. And they're just going ahead and giving people shots of stem cells that haven't really been proven to work or be safe, and they're charging people for it."

Unproven stem cell treatments can cost tens of thousands of dollars and aren't covered by insurance. Some with disposable income can at least absorb the hit. But Srivastava says he's spoken with other patients who went to great lengths to pay for stem cell treatment, taking out a mortgage on their house, "and six months later find out they're no better off than they were before."

Others have reported physical harm from stem cell injections -- from infection related to the procedure to cases of blindness. Such injuries were documented for three patients who received stem cell injections in their eyes at a clinic operated by Florida-based U.S. Stem Cell, as reported in the New England Journal of Medicine. The injured patients had been hoping to treat the progressive eye disease macular degeneration.

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Why FDA Regulation Is Important

To date, the stem cell treatment industry has remained largely unregulated. The U.S. Food and Drug Administration has been working to change that, and a recent legal decision involving Florida-based U.S. Stem Cell indicates that the FDA may be able to regulate the industry, at least in part, in the future.

In that June 2019 decision, U.S. district Judge Ursula Ungaro of Miami ordered U.S. Stem Cell operations in Weston and Sunrise, Florida, stop offering stem cell treatments. Her decision was in response to a motion that the U.S. Department of Justice filed on the FDA's behalf, seeking permanent injunction against U.S. Stem Cell and the organization's chief scientific officer, Kristin Comella. "Cell-based regenerative medicine holds significant medical opportunity, but those in this field who do not operate in compliance with the law can potentially cause serious harm to patients," acting FDA Commissioner, Dr. Ned Sharpless, said in a statement following the ruling.

The case highlights some of the very concerns some stem cell experts say consumers should be aware of when considering stem cell treatment. The FDA notes the company hadn't received approval to administer stem cells, which the FDA considers biological products, for the wide variety of serious conditions it was claiming to treat, from Parkinson's disease to ALS (or amyotropic lateral sclerosis) to chronic obstructive pulmonary disease (or COPD). Therefore, the FDA raised concerns that without proper written procedures in place, patients could be put at risk for infections.

Industry proponents say the FDA has no right to regulate stem cells, like those used by U.S. Stem Cell, which were taken from the patient's own body -- in this case, their fat tissue. "By removing a patient's own cells and injecting them where they are needed, the cells can ignite the body's healing response," Comella claims in an op-ed she wrote for The Washington Times defending U.S. Stem Cell. While the company has settled lawsuits with patients who went blind, Comella says that the media, lawyers and others have chosen to focus on a "handful of adverse incidents," rather than positive patient outcomes.

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Proceed With Caution

Proponents of regulation say that regulation of stem cell treatment -- similar to regulation of other biologic products like vaccines -- is precisely what's needed so that therapies are thoroughly evaluated before they're offered to patients. This would ensure, for example, the origin as well as the authenticity of stem cells. Experts note it's not always clear what type of cells are used in advertised therapies -- or whether they're even stem cells.

While some in the stem cell treatment industry are steadfast in the belief that the FDA is getting between patients and treatment options, other clinicians and stem cell researchers say patients should be wary of clinics that take a starkly adversarial stance against federal regulation.

Whether patients are thinking about undergoing stem cell treatments offered by hospitals and other health care organizations or standalone stem cell clinics, the advice is the same: Proceed with caution.

Check for Proper Oversight

That includes checking to make sure any experimental treatment trial has proper oversight. "The FDA would oversee any experimental trial leading to a therapy," as well as approving stem cell treatments that are at least as safe and effective as current therapies, says Dr. Irving Weissman, director of the Stanford Institute for Stem Cell Biology and Regenerative Medicine, and a former president of the International Society for Stem Cell Research.

Other questions to ask:

-- What, if any, evidence supports the stem cell therapy offered?

-- What stem cells are going to be used? And where did they come from?

Bottom line: Before you undergo any treatment, make sure you have a firm handle on what -- if any -- independent evidence backs it up. If you're overwhelmed or feel unable to sift through the case that's being made to support a treatment, seek a second opinion from a trusted, independent doctor (i.e. who isn't affiliated with the organization offering stem cell treatment), before proceeding.

"Most of the claims for stem cell therapies are lacking evidence that they work," Weissman stresses. He adds that a huge industry "has grown up around fake stem cell therapies."

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The Future of Stem Cells

Meanwhile, stem cell researchers say there continues to be reason to be hopeful for the future -- although patience is still called for when it comes to understanding and harnessing the full potential of stem cells. "It's been now 20 years since we could first grow human embryonic stem cells in a dish, and about a little over 10 years since we could make stem cells from adults that are very much like human embryonic stem cells," Srivastava says.

"We spent the last decade being able to efficiently turn those stem cells into the types desired for transplantation, and taking them through pre-clinical studies in various animal models," he adds. "Over the last several years, I think the field's hit an inflection point, where a number of those therapies are finally in clinical trials or about to be in clinical trials."

Srivastava and others say they understand the desperation some patients and families feel searching vainly for a cure that isn't yet available. But for any treatment -- experimental or otherwise -- experts say it's important to take a hard look at the evidence, the risks and alternatives. "I don't recommend," he says, "that they engage in getting therapies for which there's not good scientific basis." If what's offered isn't backed by research, consider alternatives, including non-curative therapies, where available, to manage chronic conditions, lessen symptoms and hopefully in some way improve quality of life.