With a Untitled Letter, Does FDA Set New Policy on Stem Cell Use in the United States?

Here is a newsbreaker I think.

J0182630Stem cells hold a great deal of promise in research and clinical trials are underway around the world to make that promise a reality.  However, many entrepreneurial efforts have been underway to use stem cells immediately, usually be extracting one’s own stem cells and then injecting them back in the body for therapeutic treatment.  One such company is Regenerative Sciences, Inc. which, from its own Web site is described as follows:

Regenerative Sciences Inc. is focused on the development of Regenexx™, a breakthrough non-surgical option for people suffering from various orthopedic disorders. We have developed a patent pending procedure that uses your own stem cells and blood growth factors to regenerate bone and cartilage.

Yesterday, however, a letter from Center for Biologics Evaluation and Research (CBER) posted on the FDA site, documents an untitled letter sent to Regenerative Sciences, Inc. regarding the procedure described above.  In doing so, at least to my thinking, has set a new course for stem cells in this country in that the FDA is considering them as a biologic treatment and not merely an injection of one’s own stem cells. 

Consider the following language:

Based on information posted on your website, mesenchymal stem cells utilized in your Regenexx™ procedure are drawn from a patient’s bone marrow, sent to a lab, isolated, and then grown using growth factors drawn from the patient’s blood before being inserted back into the patient. These cells are considered drugs because the therapeutic claims shown on your website demonstrate that they are intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease in man. In describing the use of mesenchymal stem cells in the Regenexx™ procedure your web site states, among other things, that: “Regenerative Sciences, Inc., has launched its patent-pending Regenexx™ procedure, which uses a patient’s own stem cells to regenerate bone and cartilage – a welcome relief for the more than 20 million Americans who suffer from painful and debilitating orthopedic conditions.” Your website also states “The procedure relieves pain and restores mobility and is a safer, less disruptive alternative to surgery”; and “With Regenexx™ almost anyone with joint pain or non-healing fractures can benefit from this groundbreaking procedure.”

For those businesses seeking to extract adult stem cells, cultivate or treat them in any way and use them in therapeutic situations, this action casts a whole new light.  As biologics such, such procedures would have to undergo FDA scrutiny and approval as a treatment as any other biologic, meaning that clinical trials should be performed before such therapies are made available to the public.  If you are harvesting your own stem cells for therapeutic interventions, this action should signal that it may be some time before you could use them.

See what else the FDA CBER had to say about the use of harvested stem cells:

Please be advised that in order to introduce or deliver for introduction a drug that is also a biological product into interstate commerce, a valid biologics license must be in effect. Such licenses are issued only after a showing of safety and efficacy for the product’s intended use. While in the development stage, such products may be distributed for clinical use in humans only if the sponsor has an investigational new drug application in effect as specified by FDA regulations (21 U.S.C. 355(i); 21 CFR Part 312). The mesenchymal stem cells utilized in your Regenexx™ procedure are not the subject of an approved biologics license application (BLA) nor is there an investigational new drug application (IND) in effect. Therefore, your implantation of the mesenchymal stem cells for which a valid license or IND is not in effect appears to violate the Act and the PHS Act and may result in FDA seeking relief as provided by law.

The mesenchymal stem cells used in your procedure are human cells, tissues, or cellular and tissue-based products (HCT/Ps) as defined in 21 CFR § 1271.3(d). Under 21 CFR Part 1271, HCT/Ps are not subject to licensure or IND requirements if certain criteria are met (21 CFR § 1271.10). However, these criteria are not met here because the mesenchymal stem cells used in your procedure are intended for non-homologous uses (21 CFR § 1271.10(a)(2); 21 CFR § 1271.3(c)).

I think that the FDA action opens up a whole new chapter.


By way of disclosure, I am part of a team recently been engaged by the International Society for Stem Cell Research to offer counsel in public affairs.  My choice to post on this topic was not influenced by that fact in any way, but by reason of the fact that I felt the FDA decision represented a substantial newsworthy policy development.

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4 Responses to With a Untitled Letter, Does FDA Set New Policy on Stem Cell Use in the United States?

  1. Greg says:

    Expanded cells grown in a bioreactor over a three week period of time should be something everybody’s concerned about, not just the FDA. The patinet’s own cells processed at the time of surgery “point of care” are not manipulated and are being use everyday in surgery around the country.

  2. Darin says:

    This is not a new policy shift by the FDA. Cellular therapies, including stem cells, have been subject to regulation by the FDA as biological products, since 1993 (see: http://www.fda.gov/cber/genadmin/fr101493.pdf). As the FDA indicated, cellular therapies are also considered HCT/Ps , subject to good tissue practice (GTP) requirements since May 2005. There are some exemptions to regulation for autologous HCT/P products. For example, if they are obtained and returned to the same patient, during the same surgical procedure. However, if they are cultured ex vivo or combined with other FDA regulated materials (drugs, biologics or devices) they would not be exempt. If they are not exempt then at a minimum they are subject to GTP requirements and based on the FDA’s letter it appears they are also subject to premarket approval meaning clinical trials under IND are required with approval as a biologic license application (BLA).
    Full disclosure: I previously worked at the FDA and was involved in the regulation of cellular therapies. I am now a regulatory consultant in this area. However, I have no knowledge of what Regenerative Sciences, Inc. is doing other than what is stated on this blog and in the FDA untitled letter.

  3. Mark Senak says:

    Darin – Thanks so much for that clarification. I did get emails from people wondering why, since the cells were from the same patient it tripped a wire, and you laid it out beautifully. I was surprised that it is not new policy, but happy for the clarification. Thanks very much.

  4. Kyle says:

    what is your opinion of this type of treatment.

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