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doi: 10.1097/01.NT.0000426342.00513.c8
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Legal Ruling Puts the Brakes on Unlicensed Stem Cell Therapies

Shaw, Gina

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ARTICLE IN BRIEF

Leading stem cell researchers agree with a legal ruling that places restrictions and FDA oversight over stem cell therapies.

Blue Horizon Stem Cells of New York City claims to offer stem cell treatments for heart disease, arthritis, diabetes, Alzheimer'sdisease, sports injuries, spinal cord injuries, stroke, and many more conditions. The Stem Cell Rejuvenation Center of Phoenix, AZ, charges patients a flat $7,600 fee to receive stem cell treatments for “a variety of conditions” — their Web site doesn't get any more specific than that. And the Stem Cell Institute of Fort Lauderdale, FL, promises treatments for multiple sclerosis, spinal cord injury, osteoarthritis, rheumatoid arthritis and other autoimmune diseases, autism, heart disease, and cerebral palsy. The price tag: between $15,000 and $20,000 — except for spinal cord injury, which one assumes costs more.

One thing these “stem cell clinics” all have in common: none of their treatments are approved by the US Food and Drug Administration (FDA). The operators argue that, since the stem cell treatments are simply “minimally manipulated” versions of the patient's own cells, these therapies don't constitute a drug or biologic that the FDA could regulate.

But a decision by a US District Court judge made last July could put the brakes on what several neurologists with expertise in stem cell research have likened to a “wild West” atmosphere. In U.S. v. Regenerative Sciences, JudgeRosemary Collyer declared that, contrary to claims made by Denver-based Regenerative Sciences, the stem cell treatments it offers for injuries and degenerative joint conditions, which it calls the “Regenexx” procedures — are subject to regulation by the US FDA.

In her opinion, Judge Collyer found that the Regenexx stem cell therapies met both the definition of a “drug” under the Federal Food, Drug, and Cosmetic Act and the definition of a “biologic” under the Public Health Service Act; she granted the FDA's request for a permanent injunction against the company.

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STEM CELL RESEARCHERS COMMENT

“I think this decision is exactly right, and I'm glad to see it,” said Arnold Kriegstein, MD, PhD, director of the Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research at the University of California, San Francisco. “It might apply the brakes to what I think is a very unethical and risky practice. There have been no trials showing efficacy for these therapies or demonstrating their safety.”

“In our field there is a strong consensus among scientists and practitioners that we need to have strict policies in place about stem cell research and therapy,” said Ole Isacson, MD, professor of neurology (neuroscience) atHarvard Medical School and director of the Neuroregeneration Research Institute at McLean Hospital. “These clinics are selling hope, or empty vials with cells in them. It's very counterproductive, and things can only be improved by stronger regulation.”

Over the past two years, the TV news magazine “60 Minutes” has conducted a series of exposés of stem cell charlatanism, focusing much of its attention on clinics based outside the U.S. Last January, they aired a program about Stem Tech Labs in Ecuador, which promised a “75 percent chance of a noticeable improvement” for an 11-year-old boy with cerebral palsy — based strictly on an over-the-phone consultation — if his parents spent $20,000 on umbilical cord stem cell treatments.

Ecuadorean clinics, of course, are beyond the reach of the FDA. But similar promises can be found here in the US.. In October, the FDA took action against Texas-based Celltech Therapeutics, touted by former Republican Governor Rick Perry, who claims the company's treatment worked wonders for his back pain. The FDA warned Celltech that its process sufficiently manipulated stem cells to make them a biologic drug subject to FDA jurisdiction, and that it was illegally marketing an unlicensed drug.

Judge Collyer's decision has been appealed, and Governor Perry has publicly called for more stem cell companies to come to Texas. At the 1st Annual Houston Stem Cell Summit, he declared, “All of you outside the state of Texas, come on in. The water's fine. We've got an amazing environment here.” He went on to say that the state has made sure that innovators won't be “hindered by exorbitant taxes, wrapped up by bureaucratic red tape or at the mercy of predatory attorneys, seeking to make steady money off extensive, drawn-out court cases.”

Frustration with the slow pace of progress in a field as promising and exciting as stem cell therapy is understandable, said ethicists Douglas Sipp, of the RIKE Center for Developmental Biology in Kobe, Japan, and Leigh Turner, of the University of Minnesota Center for Bioethics, in an opinion piece in the Dec. 7, 2012, edition of Science.

DR. OLE ISACSON: In ...
DR. OLE ISACSON: In ...
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“But if patients are to have confidence that marketed health-care products, including cell-based ones, are not only safe to take but are worth taking at all, then conformity with scientific and regulatory standards is essential,” they write. “Much of the contemporary debate concerning federal regulation of stem cells and other emerging biomedical technologies focuses on whether hurdles to market entry should be lowered. Given the efforts by various companies to bypass efficacy requirements and to promote their inadequately tested products directly to consumers, perhaps the more meaningful question is whether the regulation of stem cell–based interventions in the United States needs to be more rigorously enforced through injunctions and seizures with significant consequences for violators who, by their actions, jeopardize public health and patient safety; undermine trust in the integrity of the health-care system; and risk damaging the field of stem cell research.”

There is some element of theplacebo effect in many stem celltreatments, acknowledges Clive Svendsen, PhD, director of the Cedars-Sinai Regenerative Medicine Institute, who in 2010 received the AAN Sheila Essey Award for his research on the use of stem cells to treat ALS.

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THE PLACEBO EFFECT

“It's been said that 50 percent of medicine is placebo,” he said. “If you get a ‘stem cell’ treatment you are excited and sure you received something important and different. This alone may boost your own immune system — or yournervous system. This placebo effectvaries across diseases. It is most pronounced inParkinson's disease, which involves lowered levels of dopamine that affect movement, motivation and reward. If the patient is about to receive a new treatment they are excited and their dopamine levels increase which in turn can reduce their symptoms — at least temporarily. It's crucial for medicine to try to untangle the placebo effect in novel stem cell therapy from the effects of the cells alone. This requires well-structured, double-blinded placebo controlled trials with oversight from the FDA. Without such trials we will never know if the cells are actually working.”

“People are very vulnerable. As soon as you say the words ‘stem cells,’ their eyes just light up. It's like snake oil 70 years ago. And while I think we can all agree that mesenchymal stem cells do something above placebo that is significant in some well controlled trials (perhaps releasing growth factors or modulating the immune response), they don't seem to survive. They are there in the body for a week or so, do something for that time and then disappear. These early therapies will probably be superseded or augmented by a cell type that can truly regenerate or repair tissue. However, there's no evidence right now, with any of the adult stem cell therapies, that that is happening and they should not be sold as such.”

True innovators in the field, said Dr. Isacson, are working with the FDA to find ways of developing safe, effective products and methods. “I was the president of the Cell TransplantSociety — a section of The Transplantation Society — when working with the Cell Therapy Liaison group. These people are the true heroes in stem cell therapy, trying to find reasonable ways to establish good procedures for commercializing this technology when it is ready.”

“Those of us doing stem cell research are very concerned that the public not be misled into thinking that these so-called therapies are scientifically credited,” Dr. Kriegstein said. “They're not. They may have risks associated and they have no demonstrated efficacy. I strongly feel they have no place in modern medical therapy.”

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MAKING STEM CELLS MORE ACCESSIBLE TO RESEARCHERS

A new collaborative venture called StemBANCC, led by Oxford University and drugmaker Roche Holding AG, will bring together ten drug companies and 23 universities to create one of the largest repositories of adult stem cells in the world.

Announced during the first week in December, the $73 million initiative (funded in part by the European Commission) will store induced pluripotent stem cells derived from the skin cells of some 500 patients suffering from a number of different diseases; they will use the iPS cells to recapitulate the disease in vitro—a particularly important means of studying neurologic disease, in which scientists usually cannot access the cells involved during a patient's lifetime without an extremely invasive brain biopsy.

Ole Svendsen, PhD, director of the Cedars-Sinai Regenerative Medicine Institute, praised the initiative, noting that the California Institute of Regenerative Medicine is also gearing up to establish a similar repository in California, also involving a partnership between industry and academia. “I met the guy [neurologist and geneticist Zameel Cader, a leader of the project] in Oxford a few months ago. It's a big undertaking,” he said. “Managing all of these stem cell lines is a tremendous task and so many people are going to be using these, so it's important to have banks.”

—Gina Shaw

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FOR FURTHER READING:

• Sipp D, Turner L. Policy Forum: U.S. regulation of stem cells as medical products. Science 2012; 338(6112):1296–1297.

Neurology Today archive on stem cell research: http://bit.ly/ZJqZkP.

• The U.S. v. Regenerative Sciences ruling: http://1.usa.gov/VO8vZA.

©2013 American Academy of Neurology

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