Judge Confirms FDA Regulation of Stem Cell Industry

A federal judge ruled recently that the Food and Drug Administration has the right to regulate stem cell companies that endanger the lives of their patients. Justice Ursula Ungaro ruled that the FDA could proceed with a lawsuit against a stem-cell company based in Florida. The company had used the fat of patients to create a stem cell treatment. The procedure is reported to have blinded at least four patients in various clinics nationwide who had used the company’s treatment. The clinics injected the stem cells into the eyes of the patients and of course, bad things happened.

What is regenerative medicine

The stem cell industry has been growing steadily in recent years. Stem cell therapy is just one form of regenerative medicine which is being used treat patients who aren’t responding to current medical treatments. Many medical treatments only treat the patient’s symptoms. Regenerative medicine aims to treat the patient’s underlying conditions (diseases, congenital issues, and injuries) by helping the body engineer, replace and regenerate a person’s cells, tissues, and organs.

The aim of regenerative medicine is to use the body’s own repair mechanisms. Regenerative medicine, such as stem-cell therapy, includes helping tissues and organs grow in a laboratory – and inserting/injecting them into the patient.  Regenerative medicine advocates say it not only helps the patient heal better, it avoids the need to look for organ donors.

Regenerative medicine is not new. Bone marrow transplants and organ transplants have been used for years. However, Stem-cell treatments and PRP (Plasma-Rich Protein) treatments are fairly new.

Regenerative medicine generally falls into three categories:

• Rejuvenation. Here the body helps heal itself. A classic example is when your body heals itself after a cut.
• Replacement. This is the use of health tissues, cells, and organs from either a living person or someone who recently died. Regenerative medicine helps the transplant work better such as reducing the risk of a rejection of an organ or infection.
• New regeneration. The most common-type of new regeneration is the use of stem cells to help restore various parts of the body.

According to the Mayo Clinic, Stem cells have the ability to develop — through a process called differentiation — into many different types of cells, such as skin cells, brain cells, lung cells and so on.

There are different kinds of stem cell treatments. Sources include fat, blood, skeletal muscle, and dental pulp.

Why the recent decision in favor of the FDA is important for regenerative medicine

The question raised by the recent court decision is when these treatments cross the line from being medical treatments into being drugs. Generally, the FDA has the authority to regulate drugs. The FDA doesn’t normally regulate medical procedures.

The FDA claimed that lawsuits like the one brought against the Florida clinic are necessary to protect the safety and trust of patients. The FDA affirmed that regenerative medicine procedures such as stem cell therapy should be based on sound science.  Many health officials, medical associations, and scientist have been critical of how clinics promote their treatments as cures for a wide range of different disorders such as multiple sclerosis, Parkinson’s disease, erectile dysfunction, and joint pain – despite unproven science and procedures that haven’t been approved by the government.

More formally, the federal court decision rules that the FDA has the legal authority to treat stem cell products as drug products – which require FDA approval and require strict compliance with FDA rules.

According to Paul Knoepfler, a stem cell biologist at the University of California at Davis.

“There are potentially hundreds of clinics using the same model, and this ruling basically says the FDA can — assuming this is not overturned on appeal — say you are using an unapproved drug, and that’s a big deal.”

The FDA authority to regulate stem cell therapies as drugs

The FDA, through the authority of the 1938 US Food, Drug, and Cosmetic Act regulates medical drugs and devices. The FDA, as a general rule does not regulate medical procedures. Medical procedures are mostly regulated through local and state medical associations and the policies and practices of hospitals and the various specialties. The main distinction between drugs and procedures was and is that procedures are mostly individual in nature while drugs are produced on a mass scale. Generally, the individual patient signs an informed consent to a procedure after the physician explains all the known risks. Patients generally rely on the FDA to fully test and study the medications doctors prescribe before the prescriptions are made.

In 2006, the FDA changed its regulatory language.

“The FDA, without public commentary, altered a single word in its regulatory language regarding cell and tissue based therapies that moved their focus from protecting the public from communicable disease transmission to asserting authority over virtually all therapies using autologous cells and tissue In effect, the Agency now claims regulatory authority over a broad category of medical procedures.”

The FDA now claims it has regulatory authority over autologous platelet rich plasma (PRP) procedures and adult autologous stem cells for promoting healing of injured tissue.

The FDA states that stem cell therapies do have the potential to provide treatments that didn’t previously exist. Stem cells have the potential to repair, restore, replace, and regenerate cells, and could possibly be used to treat many medical conditions and diseases. The FDA is, however, increasing its oversight of stem cell therapies to help protect patients from dishonest and dangerous stem cell clinics – while also encouraging research that can help many members of the public.

Generally, the FDA is examining many stem cell therapies under its Investigational New Drug Application (IND) process. The FDA, as part of an IND review, examines how the product is manufactured so that the product (stem cell therapy) can be tested for safety, purity, and potency. Generally, the FDA requires that there be sufficient data from animal studies to help evaluate any potential risks associated with product use. Safety concerns include:

• Administration site reactions
• The ability of cells to move from placement sites and change into inappropriate cell types or multiply
• Failure of cells to work as expected
• The growth of tumors
• Contamination

Currently, the only stem cell-based products that are FDA-approved for use in the United States consist of blood-forming stem cells (hematopoietic progenitor cells) derived from cord blood.

The FDA states that,

“When stem cell products are used in unapproved ways—or when they are processed in ways that are more than minimally manipulated, which relates to the nature and degree of processing—the FDA may take (and has already taken) a variety of administrative and judicial actions, including criminal enforcement, depending on the violations involved.”

The facts of the stem-cell case

The company had continued the stem cell treatment for four years due to a slow response by the FDA. Though it stopped the stem-cell injections into patients’ eyes, it did sell the treatment for use to treat other diseases and disorders including spinal cord injuries and multiple sclerosis.

In a more recent case, a patient started vomiting and fainted after receiving the Florida company’s treatment for arthritis pain.

The federal judge ordered that the Florida company stop using the procedure because there was a reasonable chance that the company would continue to violate regulations by offering the unapproved treatments.

The Florida stem cell company, U.S. Stem Cell, said it would comply with the judge’s decision though it claimed “there is substantial evidence to prove the efficacy” of the procedure. The company said it would continue offering stem cell treatments derived from bone marrow and other tissue.

U.S. Stem cell made $6.7 million in revenue during the last 12 months. It has trained 150 physicians at three clinics for its various stem cell treatments.

The FDA claimed that the Florida stem cell company “openly violated the law and endangered patients by manufacturing an unapproved, experimental drug.”  U.S. Stem cell claimed that its stem cell procedures are medical procedures and not drugs – and therefore can’t be regulated by the FDA. The judge rejected that argument. She noted that the company made many claims about the medical benefits of its fat cell treatments – such as their ability to benefit people with Parkinson’s, diabetes, and lung disease. The judge also noted that the company “boasted its therapy is superior to conventional medicine in treating these serious diseases.”

The FDA, according to the judge, had inspected the company’s clinic seven times.

The likely effect of the court ruling

The judge’s ruling, according to a University of Missouri law professor doesn’t automatically mean other companies can’t offer the same treatment. The ruling, according the law professor, does send a strong sign to the market and to other judges who will likely decide similar cases.

The question raised by the judge’s ruling is whether other companies will abide by the decision or whether they’ll take their chances the FDA won’t investigate them. Some companies are expected to continue with the fat-based procedures. Others may venture into even more questionable procedures.

Former FDA commissioner Scott Gottlieb said that the ruling does put the FDA in a firm position to challenge the fat-cell procedure and other unapproved stem cell therapies.

The Food and Drug Administration has asserted, since 2006, that it has the authority to regulate stem cell therapies and other regenerative medicine therapies such as PRP. The recent decision by a federal judge to enjoin a stem-cell company from continuing dangerous treatments only helps to confirm the FDA’s authority. As a general rule, medical companies and clinics that develop and sell stem-cell therapies, need to review their FDA compliance obligations with an experienced FDA healthcare attorney.

Contact Cohen Healthcare Law Group, PC to review whether any medical procedures or regenerative products you may be marketing potentially could be classified by FDA as drugs or medical devices. We’ll explain which treatments the FDA may regulate and what specific regulations apply to your stem cell treatment or regenerative medicine treatment, and propose recommendations to help mitigate your legal risk and regulatory exposure.