Stem Cells and the Lawsuit That May Shape Our Medical Future

Regenerative Sciences, a medical company that pioneered a procedure to treat orthopedic injuries using patients' own stem cells, is fighting the Food and Drug Administration tooth and nail over a claim that human cells should be federally regulated as drugs, in a landmark case that has far-reaching implications for the future of regenerative medicine.

At the heart of the debate is a therapy that uses stem cells derived from bone marrow to repair damaged joints. It was developed in 2005 by the Colorado-based company, which began offering it to patients around 2007, and has since gathered a raft of clinical evidence and testimony about its safety and efficacy. The FDA is questioning its legality, alleging that the stem cells it uses are more than minimally manipulated drugs and should be regulated and subject to approval as drugs. In 2008, the agency accused Regenerative of practicing medicine without a license required for the introduction of a new drug, and in 2010 sued to stop it from performing the procedure.

Despite the controversy, a number of clinics from coast to coast have licensed their physicians as recently as last month to provide the cell therapy, called Regenexx. This has led to renewed interest by consumers and would-be patients, and prompted the company to publish a sharply worded blog post on its website condemning the FDA's reasoning.

The lawsuit is "concerning for every American who considers their body not to be an FDA regulated drug factory," the blog says.

While the treatment that is the focus of the lawsuit is not used for life-threatening injuries, the company claims this case goes beyond a particular procedure to shed light on a misguided push by the FDA to establish authority over aspects of medicine never allowed it by Congress.

"We see this lawsuit as a 21st century civil rights issue that will define what control you have about the use of your own cells and tissue," said Dr. Christopher Centeno, director of the Colorado clinic, in a telephone interview. "If a loved one is dying in intensive care and a well done study shows that the patient's own cells can be used to help, does the patient get to decide to use those cells, or is that a decision for the FDA? Will the patient still be alive while we wait on Washington to issue this decision?"

Centeno said his company welcomed the lawsuit because they anticipated it would finally give them a chance to formally question the FDA on its policy. Since then, Regenexx has been formally supported by the American Association of Orthopedic Medicine, the Association of American Physicians & Surgeons, legal and medical experts, and an academic who testified that one of the FDA's own medical experts who criticized the technology had a competing device.

The academic, Michael Freeman of Oregon Health Sciences University, has also faulted the FDA for its assertion that the Regenexx procedure presents a public health risk. Since the stem cells originate from the same patient into whom they are later re-injected, the treatment poses a lesser public health risk than that associated with current common medical practices and FDA-approved drugs, Freeman says in his testimony.

Regenexx works by taking a blood sample and a bone marrow sample from a patient and separating out the stem cells via centrifuge, then re-injecting those cells directly into the injured area, where they assimilate into the bone or cartilage and begin to regenerate it. Physicians for the company have shown in clinical studies cataloged at the U.S. National Library of Medicine that the therapy produces fewer and less severe complications than the more invasive and costlier surgical procedures it helps many patients avoid.

Regenerative maintains that complying with a rule that defines stem cells as drugs would impose an unbearable administrative and economic burden to the clinic and others like it, effectively stifling the industry and causing it to slow or abandon efforts to launch such treatments. Experts say that economic motivations on both sides of the debate are certainly at play.

"If it turns out the company is correct and prevails, the FDA would not be in a position to regulate the production and delivery of patients' stem cells into themselves," said Alan Russell, founder of the McGowan Institute for Regenerative Medicine at the University of Pittsburgh and a former director of the Armed Forces Institute for Regenerative Medicine, which aims to develop new treatments for wounded soldiers. "That would remove massive commercial barriers to the implementation of this science and technology, because it would remove a lot of steps that cost a lot of time and money."

"From a business standpoint, the implications are enormous. This could become very quickly a multi-billion dollar business," Russell, named one of Rolling Stone magazine's Top 100 People Who Will Change America in 2009, also said.

The sentiment is echoed by Tommy Curry, a civil rights expert and philosophy professor at Texas A & M University.

"We have to recognize that the FDA is not simply a neutral agency. Despite advancing certain definitions that are supposed to protect the public, it's still subject to economic, social and political structures," Curry said. He cited the economic benefit to pharmaceutical companies that would be the first to develop stem cell products that fit the FDA's expanded definition of a drug, and "lock the market."

Curry noted that regulating human stem cells is a dangerous proposition, because it would also effectively deny treatment to willing patients, regardless of their ability to pay, unless approved by the FDA.

"People who have the money to heal themselves may no longer have the right to do that. That says something to the public that in some way you have to suffer, to endure illness, because of how something is classified. Once we see that, we can see why it does become a civil rights issue in the sense that, does the government have the right to regulate your own blood and cells?"

But Russell, a medical futurist who is deeply involved in tissue engineering research, a field at the forefront of regenerative medicine, and currently sits on 25 advisory boards including the FDA's, believes the agency has no ulterior motive in pursuing the complaint. "The FDA is not doing anything at all that's driven by a desire other than to protect people. The FDA sees that many of these therapies are not proven to be effective, and in many cases they're not even proven safe yet," he said.

Regulators have argued that the Regenexx procedure is equivalent to the administration of a drug because the stem cells that are re-injected into patients constitute an "‘article' that is intended to treat, cure, and mitigate diseases and to affect the structure and function of the patient's body," therefore fitting within the definition of "drug."

A spokeswoman for the agency declined to comment on the case.

Whatever the outcome, "the post-Regenerative world will continue to be a regulatory minefield," law firm K&L Gates wrote in a legal insight memo concerning the monumental case.