Identifying the Risks of Unproven Regenerative Medicine Therapies

Peter W. Marks; Stephen Hahn

doi:10.1001/jama.2020.9375

Viewpoint

June 17, 2020

Identifying the Risks of Unproven Regenerative Medicine Therapies

Peter W. Marks, MD, PhD¹; Stephen Hahn, MD¹

Author Affiliations Article Information

¹US Food and Drug Administration, Silver Spring, Maryland

JAMA. 2020;324(3):241-242. doi:10.1001/jama.2020.9375

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In the middle of the coronavirus disease 2019 (COVID-19) public health response, many priorities are currently competing for the attention of the US Food and Drug Administration (FDA). Some of these are directly related to the pandemic, but others have been challenging issues for years. Over the past several years, hundreds of clinics across the US have been offering unproven regenerative medicine therapies to patients for the treatment of conditions ranging from aging to arthritis to autism.¹^,2 Some of these same clinics are now offering similar unproven products for the treatment of complications of COVID-19 and are making claims that are simply not supported by compelling clinical data. Overall, the safety and efficacy of regenerative medicine products outside a narrow range of indications have yet to be demonstrated.³

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2 Comments for this article

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June 17, 2020

Need Quotation Marks

Robert Roth, MD | Retired GP

Any reason not to refer to the subject of your paper as "regenerative therapies," in quotes? Use of the quotation marks implies that you doubt the claim -- which I gather you do, and rightly so. Not using them implies that such things do exist. I don't think there is a middle ground there.

CONFLICT OF INTEREST: None Reported

June 18, 2020

Real World data Essential For Determining Safety and Efficacy of "Regenerative Treatments"

Gerard Malanga, M.D., Clin Prof Rutgers NJ | DataBiologics

It was with great interest that I read the article by Drs. Marks and Hahn: "Identifying the Risks of Unproven Regenerative Medicine Therapies". As a clinician and clinical researcher in "regenerative treatments" for orthopedic conditions, I fully agree that this area needs to be more done by clinicians to determine the safety and potential efficacy of these treatments. There is a great deal of lab and animal studies to support the potential benefit of these treatments for common diagnoses such as knee osteoarthritis and tendinopathies. In additional, there is an increasing number of clinical studies that demonstrated both safety and efficacy of these treatments for orthopedic conditions with Phillipe Hernigou publishing data with 20 year follow up on the use of bone marrow MSCs in various orthopedic conditions including a group treated with bone tumors. While there have been isolated severe adverse cases, these have occurred in patients either receiving an inappropriate procedure (i.e. ocular injection of adipose) or improperly manufactured products such as umbilical cord stem cells or exosomes which have no animal or clinical evidence for use.

I do not believe the haphazard approach of individual patients or clinicians reporting adverse events will ultimately assist the FDA or general public in making it "possible for the FDA to gain a better understanding of the potential spectrum of adverse events associated with these therapies." Rather, having any clinician wishing to provide these therapies to patients outside of a clinical trial i.e. gathering real world data required to collect safety and efficacy data by an independent third party platform, would quickly and transparently produce the data needed for everyone to make informed decisions on this controversial topic. This historically has been costly and time-consuming and is why I and colleagues created a database registry available to all thoughtful clinicians to collect and share this data with their patients and others. This national database registry will better allow for the "mission to which the FDA is committed: promoting and protecting the public health" and allow for thoughtful application of these treatments.

CONFLICT OF INTEREST: I am a Founder and Partner of DataBiologics a national database registry for Regenerative procedures for orthopedic conditions.

Identifying the Risks of Unproven Regenerative Medicine Therapies

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