FDA Should Tread Carefully with Stem Cell Regulation

(Editor’s note: This week FDA commissioner Scott Gottlieb said the agency would crack down on clinics that offer experimental stem cell treatments. While acknowledging that stem cells offer “significant promise” for treating chronic pain and other chronic conditions, Gottlieb said some clinics were preying on sick people desperate to find cures.

A. Rahman Ford has received stem cell treatment and has been closely following developments in stem cell research and regenerative medicine.)

By A. Rahman Ford, Columnist

On its face, FDA Commissioner Gottlieb's statement seems like a reasonable approach to regulating stem cell therapies.  He emphasizes the need to balance costs and benefits, and the need to protect vulnerable consumers from exploitation. 

I think all can agree with the Commissioner when he states that "these technologies hold the potential to significantly alter the course of a broad range of diseases."  These diseases are often accompanied by intense physical pain that pharmaceuticals cannot relieve.

Specifically, the Commissioner makes three heartening observations. First, he acknowledges the "significant promise for transformative and potentially curative treatments" offered by regenerative medicine.  This is a promise that mainstream pharmaceutical medicine has yet to fulfill, and one that those suffering from chronic pain need addressed immediately. 

Second, it is without question that regulatory clarity and the resolution of legal uncertainty regarding these therapies' use can be a good thing.  If there are in fact an irresponsible "select few," or a minority of unscrupulous, exploitative "bad actors" as the Commissioner claims, then legal action by the FDA against them is reasonable. 

fda commissioner scott gottlieb

fda commissioner scott gottlieb

Finally, the Commissioner seems to implicitly recognize how critical it is for the FDA to respect the distinction between medical products subject to agency oversight, and those which fall under the "practice of medicine," a distinction which Commissioner Gottlieb acknowledges is fraught with "close calls."

That said, as an advocate for patients seeking stem cell therapies, upon reading Commissioner Gottlieb's statement I am overall circumspect.  It is well known that the FDA has a rather unsettling history of issuing regulations that unduly restrict patients' access to stem cell therapies, specifically autologous stem cell therapies.  These are therapies that use cells harvested from one person, are minimally manipulated, and then administered to the same person. 

I've previously made my position on this "personal" stem cell therapy known, particularly the privacy implications involved. 

Restrictions such as the unduly burdensome "minimally manipulated" federal standard and the "same basic function" requirement are clear evidence of an intent to restrict.  So, the fact that Commissioner Gottlieb's statement reiterates the "same basic function" standard as a factor in requiring that a stem cell therapy be approved through a clinical trial, rather than it being part of the "practice of medicine," seems to indicate more of the same federal overreach.

Make no mistake, the potential dangers of stem cell therapy are real, and proactive effort to protect patients is far better than any reactive effort.  However, the potential dangers should not be overstated or exaggerated ostensibly toward the end of more unnecessary restrictive regulation. 

The FDA has a history of doing this.  For example, the New England Journal of Medicine published a commentary in March by then FDA Commissioner Robert Califf arguing that, aside from a few indications, the clinical use of autologous stem cell therapies has not been proven effective and can even be dangerous. 

Not only did the article ignore years of clinical data from medical practitioners, the one cited example of the danger of autologous stem cells was actually an example of allogeneic stem cell use -- stem cells from another person!

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Thus, when the Commissioner asserts that the FDA will "aid in the effort to bring novel therapies to patients as quickly, and as safely, as possible," it simply belies history.  Likewise, overtures made toward any potential regulations being congruent with the 21st Century Cures Act are dubious.  The spirit of the Cures Act is clear; it calls for the "accelerated approval for advanced regenerative therapies." 

More federal regulation rarely, if ever, leads to acceleration of anything.  In fact, it almost always tends to slow things down.  Thus, unnecessary and unreasonably burdensome regulation by the FDA could contravene the will of Congress, and thus the will of the American people.

Texas Legalizes 'Personal' Stem Cell Therapy

Furthermore, the FDA's prospective regulatory guidance must be viewed in the context of recent events in Texas.  On June 13, Texas governor Greg Abbot signed HB 810 into law, which made Texas the first state to legitimize the use of personal stem cell therapies statutorily. The signing of the bill was celebrated not only by stem cell advocates, but by the countless Americans who suffer from chronic, debilitating conditions for which the current medical services delivery model can offer only surgery and medication.  For many, it was a monumental step forward toward fulfilling the promise of regenerative medicine and realization of true health care.  

However, the FDA may have seen this move as reinforcing a "wild west" stem cell landscape, a landscape which it believes it must police.

All of us, including FDA officials, should be reminded that on February 28, at President Trump's first State of the Union speech (and on Rare Disease Day), the President took note of Sarah Hughes, a young attendee who had used her own stem cells to successfully treat Pompe’s Disease. The therapy normalized her immune system, alleviated her symptoms and helped reduce her medications from 22 to 8.

We must also remember the promise President Trump made to America's military veterans, many of whom suffer from painful, debilitating conditions that may be treated or cured by stem cells.  President Trump cares deeply about veterans' health.  He recently signed the Veterans Appeals Improvement and Modernization Act, which streamlines the process of veterans appealing claims over disability benefits.  He also signed a bill that will let more veterans bypass the Department of Veterans Affairs and instead receive treatment from private doctors. 

Finally, he signed legislation approving new tools to expand the VA's existing Telehealth Services, so veterans can schedule appointments and have video consultations from their mobile phones.  It seems obvious that President Trump would never support restricting veterans' access to the medical care they need, including stem cells.

I believe that President Trump, through his devotion to our veterans, the Congress through the Cures Act, and the American people through their need for medical alternatives, would strongly disagree with any unreasonable curtailment of stem cell therapies. 

The FDA must not defer to the opinion of "industry," and must prioritize the needs of Americans like Sarah Hughes and our suffering wounded warriors.  People are in pain and pills can’t always help them.

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A. Rahman Ford, PhD, is a lawyer and research professional. 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.

Dr. Ford is not affiliated with any stem cell treatment provider. He suffers from chronic inflammation in his digestive tract and is unable to eat solid food.

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.