Using your own stem cells, tissue and body parts without FDA interference should be a no brainer and a slam dunk, but it isn’t. I mean it’s your own body. How can the federal government interfere with your privacy and autonomy right to use parts of your own body as a treatment? The short answer is they can and if the FDA gets its way, future patients are not going to be able to use their own cells and tissue as tens or hundreds of thousands of patients, including breast reconstruction patients, have been doing for years. Here is how the FDA is going to stop you from using your own stuff (stem cells and tissue like fat, or what the FDA calls “Human Cells, Tissues and Cellular and Tissue-Based Products” or “HCT/P’s as the federales call it.) But first some short regulatory history
How the FDA thinks of your body parts
Until the 1990’s, removing and reinserting body parts wasn’t regulated by the FDA, other than making sure that it was collected and maintained in a safe and sterile way. But then stem cells started to become a popular research field, and all the hype started about the magical therapeutic power or potential of stem cells. So in the late 1990’s the FDA started making noises about regulating stem cells and other human tissue. That would include both your own stems cells (called in medical and regulatory parlance “autologous”, and someone else’s stem cells or tissue (“allogenic”).
In 2005, the FDA published its final rules concerning HCT/P’s. (Here is the link to 21 CFR 1271, for the legally curious. (http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?CFRPart=1271)
The regulations created three different regulatory pathways for HCT/P’s; some were considered “new drugs” which required full FDA approval, which is a hideously expensive and unreasonably long endeavor. (This is sometimes referred to as the 351 pathway); some just required that the facility using the stem cells or other tissue register with the FDA (sometimes referred to as the 361 pathway), and if the stem cells or other tissue was removed and reinserted into the person in the “same surgical procedure,” the FDA didn’t regulate it at all because it was the practice of medicine which supposedly the FDA doesn’t regulate.
The FDA’s 2005 regulations introduced two biologic/analytical binary concepts which it uses as criteria for determining which of the three regulatory pathways applied to a particular use of an HCT/P: Homologous vs. non homologous and minimally manipulated vs. more than minimally manipulated.
To oversimply, a use of an HCT/P is homologous if the use of the material in the donor location is the same as in the recipient location. When the FDA regulations were first put out in 2005, there was some vagueness or flexibility in what constituted the same or homologous use. The regulations stipulated that if the use was homologous, the use might only require registration under the 361 regulatory pathway (and if a number of other requirements were met). But if the HCT/P use was non-homologous (meaning that the use the cell had in the donor location was different from the intended use in the recipient location), then regulatory magic turned your HCT/P into a 351 new drug with the aforementioned hideously expensive and unreasonably long consequences. Seems crazy, but that was the reg.
Now you usually can’t just take stem cells out of your body; they have to be separated from fat, bone marrow, or blood. That means after you take out the material, the stem cells or other material is separated, using either a chemical agent or a mechanical process (like a centrifuge). Usually there is also other processing needed before the material is reimplanted.
The processing of the material gave rise to the second binary concept, minimal manipulation vs. more than minimal manipulation of the HCT/P. If the cells are not more than minimally manipulated, the procedure could be done with just 361 facility registration (assuming homologous use and several other technical requirements set out in the 1271.1 (Here’s the link to the regulation if you’re turned-on by that kind of thing. http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?fr=1271.10 )
But if the cells were more than minimally manipulated, then you are thrown back to the new drug path. Remember, we’re talking about your own stuff. Under the rule, there was some ambiguity as to what was or wasn’t minimal manipulation. Back when the rules first became effective, I had a client who wanted to take out fat, separate the mesenchymal stem cells and reinsert the cells back. To me, it seemed legal under the rules. I called the FDA a couple times to make sure I was right, and everyone at the agency I talked to agreed. But that was then.
Since that time, there has been an explosion of stem cell clinics taking fat from people and reinjecting the stem cells. Worse from the FDA’s point of view, many of these clinics were making all kinds of miraculous cure claims. Problem was there was no proof that the claims were true for most of the diseases or problems claimed to be cured or helped by stem cells. That made the FDA unhappy, which is bad thing. (Trust me, I know how bad it is when the FDA gets unhappy).
Long story short: the FDA decided to clamp down on all of these clinics, and used two of its most effective tactics.
First, it sent cease and desist letters (what the FDA calls a “warning letter”) to a couple of the most visible offenders. (Hit the big guys first to scare-off everyone else). The FDA publishes all warnings letters, and that got the industry’s attention.
Second, in the last year or two, the FDA issued four draft guidance documents “clarifying” the meaning the two key terms homologous/non homologous use and more than minimally manipulated (or not). But the FDA didn’t just clarify these terms; it rewrote the rule to knock-out some of the most popular and effective medical procedures, perhaps most importantly, the most popular post mastectomy breast reconstruction procedure with flap surgery.
No point getting too much in the weeds of how the feds did it (see my last post if the legal weeds is your thing.
But here is the most egregious example that shows the abject stupidity of what the FDA is trying to do:
Flap reconstructive breast surgery takes fat (usually belly fat) and builds up the breast. Under the guidance documents, the main function of fat is structural. The main function of a breast (in a woman) is lactation. (Some presenters of the female persuasion who presented at the stem cell public hearing were really annoyed about that characterization). Lactation is a different function than the function of belly fat (structural). Therefore the use the flap procedure is non-homologous, which means the use has to obtain full FDA approval before it can be used outside of FDA approved clinical trials. Many of the presenters used this as an example of why the draft guidelines are wrong. (more details in my last post:)
The fact that tens of thousands of women have had this procedure and are walking around feeling better about themselves is irrelevant to the FDA.
I’d like to meet the idiot who came up with this. Better still, I’d like to put him/her/them in a room with a few mastectomy patients who need the flap surgery, and let them explain to the patients why their belly fat and breasts are any of the FDA’s business.
Remember when I said that the FDA told me that docs can take fat out, separate the stem cells and reimplant them without FDA oversight if it was done during the same surgical procedure? Well that’s out the window now. Under the guidance documents, separating the stem cells from the fat constitutes “more than minimal manipulation” of the HCT/P and requires full-on FDA approval because your stem cells are now an unapproved drug and they can’t be reinjected into you without being approved by the FDA.
I get that the FDA is concerned about clinics making unsubstantiated claims. There are also a few well-publicized incidents of harm, most notably some clinic which apparently let a nurse practitioner inject stem cells in the eyeballs of patients. What idiot decided that was a good idea? Still, the remedy to that kind of problem is or could be a combination of civil actions by the patients, criminal prosecution, professional licensure proceedings, or state action to shut the place down. As to claims, The FTC has jurisdiction to deal with false claims issue, as does the state under state consumer deceptive trade practice laws.
The point is that there are a lot of options for dealing with places which do crazy things, harm patients, or make outrageous claims. The solution shouldn’t be that FDA closes down the entire autologous transplant industry because of some exceptional bad examples. Describing this as overkill is an understatement.
So here’s what needs to be done. The FDA has to rescind the draft guidance documents and start over. In the interim, the FDA should get the hell away from my stem cells and other body parts and yours too, at least if we want to take them out and put them back in our own bodies.
What can you do about it? Plenty
The public hearing of the FDA’s stem cell guidance documents ended last Tuesday, September 13, 2016, but the public comment period is open until September 27th.
If you think you might ever need to use your own stem cells or other body parts in the future, or if you know anyone how might need them, or if you think that what the FDA is doing is a bad idea, then write, fax or email the FDA and tell them. Here are some possible points:
- Withdraw the four HCT/P guidance documents
- Get out of the business of regulating a person’s use of his own body parts
- Any opinions you might have about where they should place their draft guidance documents, or such other opinions you might have on this regulatory exercise, mindful of the rules of polite discourse, based on your discretion and/or temperament.
Maybe if a few hundred thousand people contact these jokers, they might get the message.
Where to send your comment?
Here is the information I found about sending comments:
“Submit written/paper submissions as follows: • Mail/Hand delivery/Courier (for written/paper submissions): Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD 20852. • For written/paper comments submitted to the Division of Dockets Management, FDA will post your comment, as well as any attachments, except for information submitted, marked and identified, as confidential, if submitted as detailed in “Instructions.” Instructions: All submissions received must include the Docket No. FDA-2015-D-3719 for “Draft Guidances Relating to the Regulation of Human Cells, Tissues, and Cellular and Tissue-Based Products; Rescheduling of Public Hearing; Request for Comments.”
If you want to go the email route: do one or both:
Or go to Federal eRulemaking Portal: http://www.regulations.gov.
FYI: here is the sub heading from the eRulemaking Portal:
Make a difference. Submit your comments and let your voice be heard.
That’s good advice.
Here is the FDA Federal Register Notice about the Hearings, where you can read the written comments, and more details on submission of comments.
If you want more details about the issue or love the weeds, here is a link to all my recent posts on this topic. They’re in reverse chronological order, so start with the last one “Are stem cells over?”
(To Clinton Miller, I hope we can do you proud.)