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Recap: FDA Issues New Guidelines for Gene, Stem Cell Therapies

Nov 29, 2017

malangaGerard A. Malanga, MD, FAAPMR
New Jersey Regenerative Institute

On November 16, 2017, the Food and Drug Administration (FDA) released new guidelines to expedite the “introduction of treatments involving human cells and tissues, including gene therapy.” I recommend that those of you who have been providing regenerative treatments in the form of platelet rich plasma and/or bone marrow and adipose derived stem cell therapies, read the article below to learn more about this important topic.

These guidelines stress that any type of stem cell therapy must be performed on the same day as the harvesting of the cellular products (i.e., the time of bone marrow or adipose aspiration and should not be processed with more than what is considered “minimal manipulation”). This means that the inherent structure of the harvest material cannot be altered, such as what occurs during enzymatic digestion of adipose tissue. The guidelines stress requirement for homologous use, which requires the cellular material be injected into areas that performed the “same basic function” of the harvest material. 

The FDA guidance has raised concerns regarding how many practitioners have been using adipose tissue for various conditions, which are felt to be non-homologous use. In addition, significant concerns were raised regarding the use of specialized tissues such as amniotic and umbilical cord tissue. Although the regenerative medicine area remains an exciting area that needs further support of research, AAPM&R members who provide regenerative treatments must remain compliant with the current guidelines and provide patients with evidence-based information regarding the various regenerative treatments, with language regarding these treatments be compliant with the FDA guidance.

ORIGINAL ARTICLE:

Published in the American Medical Association's newsletter, AMA Morning Rounds®

The New York Times (11/16, Kaplan, Grady, Subscription Publication) reports the FDA on Thursday issued new guidelines to expedite the “introduction of treatments involving human cells and tissues, including gene therapy.” The agency also said it will crack down on “rogue clinics offering dangerous or unproven versions of those treatments.” FDA Commissioner Scott Gottlieb, MD, said concepts like gene therapies are “no longer the stuff of science fiction, but rather, real-life science where cells and tissues can be engineered to grow healthy, functional organs to replace diseased ones; where new genes can be introduced into the body to combat disease; and where adult stem cells can generate replacements for cells that are lost to injury or illness.” According to the guidelines, promising gene and cell therapies to treat unmet medical needs and serious illnesses may qualify for expedited review. The agency will still “require clinical trials, but it is promising a faster process, as required by Congress under the 2016 21st Century Cures Act.”

 The AP (11/16, Perrone) reports the FDA outlined a strategy to increase oversight on the hundreds of private clinics offering stem cell procedures that have opened across the nation in the past decade. Many of these businesses promote stem cell injections to treat diseases that “can cost $5,000 to $50,000, but there’s little research that such procedures are safe or effective.” FDA officials said they will focus their enforcement efforts on “bad actors” in the industry, who “inject stem cell mixtures into the bloodstream, nervous system or eyes.” Dr. Gottlieb said on a conference call with reporters, “We’re going to be prioritizing places where we see products – not just being promoted inappropriately – but putting patients at potential risk.” He added that the agency will use discretion in overseeing lower-risk procedures.

 STAT (11/16, Joseph) reports that the FDA said it will “use discretion when enforcing such regulations over the next three years to give clinics a chance to comply with them and apply for FDA approval for their products, but that the agency will crack down on products that could be dangerous.” However, some experts in favor of tougher regulations of clinics “have said the FDA needs to be taking more enforcement actions now and that the three-year transition period could allow the clinics to keep putting patients at risk.”