By A. Rahman Ford, Columnist
In August, FDA Commissioner Scott Gottlieb, MD, signaled that his agency would move in the direction of enhanced regulation of stem cell therapies. He said the action was justified because of “unscrupulous actors” who deceive patients with what he described as “dangerously dubious products.”
Although the FDA referred to its prospective actions benignly as “enforcement” and “protection,” the likely impact seemed obvious – safe and effective therapies that have been healing patients for years would become less available and more expensive.
Those concerns have now been made real.
This week the FDA released its final guidance on regenerative medicine and stem cell therapy, an approach that relies extensively on more regulation.
“We’re at the beginning of a paradigm change in medicine with the promise of being able to facilitate regeneration of parts of the human body, where cells and tissues can be engineered to grow healthy, functional organs to replace diseased ones; new genes can be introduced into the body to combat disease; and adult stem cells can generate replacements for cells that are lost to injury or disease,” Gottlieb said in a statement.
“We’re adopting a risk-based and science-based approach that builds upon existing regulations to support innovative product development while clarifying the FDA’s authorities and enforcement priorities. This will protect patients from products that pose potential significant risks, while accelerating access to safe and effective new therapies.”
Unfortunately, from both a patient and policy perspective, the FDA’s guidance is distinctly retrograde, drifting backward against a cosmic tide of scientific discovery that should be propelling post-modern medicine forward. Not only are the FDA’s actions potentially detrimental to the health of Americans who suffer from chronic illnesses that could be treated or even cured by stem cells, they could cripple the entrepreneurship, ingenuity and cost-effectiveness of regenerative medicine.
For the next 36 months, Gottlieb says FDA will adopt a “risk-based approach” in enforcement of the new rules, “taking into account how products are being administered as well as the diseases and conditions for which they are being used.” While this grace period seems reasonable, the final guidance makes clear that the FDA will prioritize the oversight of clinics that deliver stem cells via intravenous infusion, in part because “use of these unapproved products may cause users to delay or discontinue medical treatments that have been found safe and effective.”
But intravenous stem cell administration has been safely used to treat patients suffering from painful autoimmune diseases like multiple sclerosis. I have personally had intravenous stem cell therapy, and found it to be both safe and effective.
‘Minimal Manipulation’ Test Tough to Pass
While well intended, the FDA’s guidance suffers from unduly restrictive definitions of “minimally manipulated” and “homologous use” -- key standards that will determine the availability of future stem cell therapies.
Stated simply, stem cells that are “minimally manipulated” will not need to be approved by the FDA via clinical trial. But that’s a tough test to pass and would seem to rule out mesenchymal stem cells, which help reduce inflammation in orthopedic and autoimmune conditions, conditions that cause unbearable pain for countless Americans. The FDA’s conception of “minimally manipulated” is simply too limited.
The “homologous use” definition is likewise flawed. Even if a stem cell product passes the “minimally manipulated” test, the cells used must “perform one or more of the same basic functions in the recipient as the cells or tissues performed in the donor.” Under this definition, blood stem cells can be transplanted into a person with a disorder affecting their blood system, but cannot be used to repair damaged tissue, as in the case of a child’s cerebral palsy or a wounded veteran’s traumatic brain injury.
However, in a cruel twist, adipose (fatty) tissue used for cosmetic procedures like breast reconstruction and augmentation do satisfy the “homologous use” test and do not require a clinical trial.
Essentially, under these guidelines, the FDA strong-arms the child with cerebral palsy and the war veteran to the back of the medical bus, while giving breast augmentation the VIP treatment and ushering it to the front. Sorry FDA, big breasts are not more important than curing diseases.
FDA Should Respect Privacy Rights
Additionally, as I have written about previously, Americans have a constitutionally-protected privacy right in their own cells. The FDA’s new rules completely ignore this right. It is a basic right that emanates from established Supreme Court case law and fundamental principles of personal liberty and autonomy, and protections against undue intrusions upon bodily integrity.
One’s body and one’s health are indeed intimate and personal matters that federal agencies must respect. Instead of being overly preoccupied with a cell’s “same basic function,” the FDA needs to be concerned with a person’s “fundamental basic right” to use their own cells as they see fit.
In fact, one could easily argue that sufficient government regulation and patient protections already exist in federal and state law, as well as medical ethics boards. The truth is that additional federal regulations are unnecessary, unduly burdensome, and infringe upon the sanctity of the physician-patient relationship and our right to control our own bodies.
Commissioner Gottlieb’s declaration that patient safety is of paramount concern is a pleasure to hear. However, in practice, the FDA’s regulatory efforts will likely serve to retard medical innovation and force Americans to travel abroad for life-saving stem cell therapies. They also preserve a “clinical trial” approach to treatment that, when applied to stem cells, has proven itself to be unduly burdensome, unreasonably slow, and unbelievably expensive.
Ultimately, what is abundantly clear is that the American people have spoken. Their voices are filled with pain and they must be heard. Retrograde stem cell policy is inimical to curing disease and is an affront to the elimination of pain.
A. Rahman Ford, PhD, is a freelance researcher and writer on the issues of politics, policy and health. He is a graduate of Rutgers University and the Howard University School of Law, where he served as Editor in Chief of the Howard Law Journal. He earned his PhD at the University of Pennsylvania.
Rahman lives with chronic inflammation in his digestive tract and is unable to eat solid food. He has received stem cell treatment in China.
The information in this column should not be considered as professional medical advice, diagnosis or treatment. It is for informational purposes only and represent the author’s opinions alone. It does not inherently express or reflect the views, opinions and/or positions of Pain News Network.